Literatur

Zuletzt geändert: 16.12.2024 https://kinderkrebsinfo.de/doi/e8952

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Autor(en)	Titel	Quelle
Vanuytsel LJ, Bessell EM, Ashley SE, Bloom HJ, Brada M	Intracranial ependymoma: long-term results of a policy of surgery and radiotherapy.	Int J Radiat Oncol Biol Phys 1992, 23: 313
Ninety-three patients with primary intracranial ependymoma were treated at the Royal Marsden Hospital, between 1952 and 1988, with postoperative radiotherapy. The survival probability at 5, 10, and 15 years was 51%, 42% and 31%, respectively, and the corresponding progression free survival (PFS) probability, 41%, 38%, and 30%. Tumor grade was the single most important prognostic factor for survival and PFS with gender of lesser prognostic significance. Treatment parameters were stratified for grade. In patients with low grade tumors survival and PFS were better following complete macroscopic excision compared to incomplete surgery. The extent of resection had no significant influence on survival or PFS in patients with high grade tumors. Extent of irradiation did not influence PFS, irrespective of tumor grade, while patients with high grade tumors had marginally better survival following extensive irradiation compared to more limited radiotherapy. The main problem in the treatment of ependymoma remains local progression which was the cause of death in all but two patients. New treatment strategies should focus on improvement of local control, especially in incompletely resected low grade tumors and all high grade tumors. The use of spinal irradiation is unlikely to significantly improve treatment results.
van Valen F, Winkelmann W, Burdach S, Göbel U, Jürgens H	Interferon gamma and tumour necrosis factor alpha induce a synergistic antiproliferative response in human Ewing's sarcoma cells in vitro.	J Cancer Res Clin Oncol 1993, 119: 615
van Valen F, Hanenberg H, Jürgens H	Expression of functional very late antigen-alpha 1-alpha 2-alpha 3 and -alpha 6 integrins on Ewing's sarcoma and primitive peripheral neuroectodermal tumour cells and modulation by interferon-gamma and tumour necrosis factor-alpha.	Eur J Cancer 1994, 30A: 2119
van Valen F, Kentrup-Lardong V, Truckenbrod B, Rübe C, Winkelmann W, Jürgens H	Regulation of the release of tumour necrosis factor (TNF)alpha and soluble TNF receptor by gamma irradiation and interferon gamma in Ewing's sarcoma / peripheral primitive neuroectodermal tumour cells.	J Cancer Res Clin Oncol 1997, 123: 245
van Dongen J, Seriu T, Panzer-Grumayer E, Biondi A, Pongers-Willemse M, Corral L, Stolz F, Schrappe M, Masera G, Kamps W, Gadner H, van Wering E, Ludwig W, Basso G, de Bruijn M, Cazzaniga G, Hettinger K, Berg van, Hop W, Riehm H, Bartram C	Prognostic value of minimal residual disease in acute lymphoblastic leukaemia in childhood.	Lancet 1998, 352: 1731
van den Heuvel-Eibrink MM, Graf N, Pein F, Sandstedt B, van Tinteren H, van der Vaart KE, de Kraker J	Intracranial relapse in Wilms tumor patients.	Pediatric blood & cancer 2004, 43: 737
BACKGROUND: In children with nephroblastoma, recurrence with metastases in the central nervous system is rare. Recently, previous reports (NWTSG and UKCCSG) reported brain metastases with an incidence of respectively 0.5% and 0.6% in Wilms tumor (WT) patients (respectively n = 30/5,852 and n = 7/1,249). PROCEDURE: We retrospectively investigated the incidence and survival of patients with central nervous system relapse in WT patients, treated according to the consecutive SIOP protocols 1, 2, 5, 6, 9, and 93-01. All children with WT from 1971 until 2000 were enrolled in the study (3,040 eligible patients). Specimens at diagnosis and if possible at relapse were centrally reviewed. Patients with renal neoplasms other than WT were excluded. RESULTS: CNS relapse was documented in 14 patients (0.5%). Median time to CNS relapse was 16 months (3-69). The occurrence of relapse was not associated with specific histological subtypes. In seven patients intracranial metastases occurred at first relapse, of which two were isolated relapses. In five patients no treatment was started because of the poor condition of the patient, the other nine cases were treated with (a combination of) chemotherapy (n = 6), surgery (n = 4), and radiotherapy (n = 6). CONCLUSIONS: CNS relapse in WNT is rare. In contrast to reports of other Wilms tumor study groups, although four patients reached (local) CR, the SIOP registry showed that eventually none of the documented WT patients survived.
Vassal G, Fitzgerald E, Schrappe M, Arnold F, Kowalczyk J, Walker D, Hjorth L, Riccardi R, Kienesberger A, Jones KP, Valsecchi MG, Janic D, Hasle H, Kearns P, Petrarulo G, Florindi F, Essiaf S, Ladenstein R	Challenges for children and adolescents with cancer in Europe: The SIOP-Europe agenda.	Pediatr Blood Cancer 2014 Apr 7
In Europe, 6,000 young people die of cancer yearly, the commonest disease causing death beyond the age of 1 year. In addition, 300,000-500,000 European citizens are survivors of a childhood cancer and up to 30% of them have severe long-term sequelae of their treatment. Increasing both cure and quality of cure are the two goals of the European paediatric haematology/oncology community. SIOPE coordinates and facilitates research, care and training which are implemented by the 18 European study groups and 23 national paediatric haematology/oncology societies. SIOPE is the European branch of the International Society of Paediatric Oncology and one of the six founding members of the European Cancer Organisation. SIOPE is preparing its strategic agenda to assure long-term sustainability of clinical and translational research in paediatric malignancies over the next 15 years. SIOPE tackles the issues of equal access to standard care and research across Europe and improvement of long term follow up. SIOPE defined a comprehensive syllabus for training European specialists. A strong partnership with parent, patient and survivor organisations is being developed to successfully achieve the goals of this patient-centred agenda. SIOPE is advocating in the field of EU policies, such as the Clinical Trials Regulation and the Paediatric Medicine Regulation, to warrant that the voice of young people is heard and their needs adequately addressed. SIOPE and the European community are entirely committed to the global agenda against childhood cancers to overcome the challenges to increasing both cure and quality of cure of young people with cancer. Pediatr Blood Cancer © 2014 Wiley Periodicals, Inc.
Vaz F, Hanenberg H, Schuster B, Barker K, Wiek C, Erven V, Neveling K, Endt D, Kesterton I, Autore F, Fraternali F, Freund M, Hartmann L, Grimwade D, Roberts RG, Schaal H, Mohammed S, Rahman N, Schindler D, Mathew CG	Mutation of the RAD51C gene in a Fanconi anemia-like disorder.	Nat. Genet 2010, 42: 406
Fanconi anemia (FA) is a rare chromosomal-instability disorder associated with a variety of developmental abnormalities, bone marrow failure and predisposition to leukemia and other cancers. We have identified a homozygous missense mutation in the RAD51C gene in a consanguineous family with multiple severe congenital abnormalities characteristic of FA. RAD51C is a member of the RAD51-like gene family involved in homologous recombination-mediated DNA repair. The mutation results in loss of RAD51 focus formation in response to DNA damage and in increased cellular sensitivity to the DNA interstrand cross-linking agent mitomycin C and the topoisomerase-1 inhibitor camptothecin. Thus, biallelic germline mutations in a RAD51 paralog are associated with an FA-like syndrome.
Van der Velden VH, Corral L, Valsecchi MG, Jansen MW, De Lorenzo P, Cazzaniga G, Panzer-Grümayer ER, Schrappe M, Schrauder A, Meyer C, Marschalek R, Nigro LL, Metzler M, Basso G, Mann G, Den Boer ML, Biondi A, Pieters R, Van Dongen JJ, Interfant-99 Study Group	Prognostic significance of minimal residual disease in infants with acute lymphoblastic leukemia treated within the Interfant-99 protocol.	Leukemia 2009, 23: 1073
Acute lymphoblastic leukemia (ALL) in infants younger than 1 year is a rare but relatively homogeneous disease ( approximately 80% MLL gene rearranged, approximately 70% CD10-negative) when compared with childhood and adult ALL. Several studies in children and adults with ALL have shown that minimal residual disease (MRD) status is a strong and independent prognostic factor. We therefore evaluated the prognostic significance of MRD in infant ALL. Ninety-nine infant patients treated according to the Interfant-99 protocol were included in this study. MRD was analyzed by real-time quantitative PCR analysis of rearranged immunoglobulin genes, T-cell receptor genes and MLL genes at various time points (TP) during therapy. Higher MRD levels at the end of induction (TP2) and consolidation (TP3) were significantly associated with lower disease-free survival. Combined MRD information at TP2 and TP3 allowed recognition of three patients groups that significantly differed in outcome. All MRD-high-risk patients (MRD levels > or =10(-4) at TP3; 26% of patients) relapsed. MRD-low-risk patients (MRD level <10(-4) at both TP2 and TP3) constituted 44% of patients and showed a relapse-rate of only 13%, whereas remaining patients (MRD-medium-risk patients; 30% of patients) had a relapse rate of 31%. Comparison between the current Interfant-06 stratification at diagnosis and the here presented MRD-based stratification showed that both stratifications recognized different subgroups of patients. These data indicate that MRD diagnostics has added value for recognition of risk groups in infant ALL and that MRD diagnostics can be used for treatment intervention in infant ALL as well.
Velleuer E, Dietrich R	Fanconi anemia: young patients at high risk for squamous cell carcinoma.	Molecular and cellular pediatrics 2014, 1: 9
Fanconi anemia is one of the best studied inherited cancer-prone diseases. Greatly improved protocols for hematopoietic stem cell transplantation increasingly save the lives of these young patients. However, in both transplanted and not transplanted patients, the emergence of aggressive squamous cell carcinoma represents a major medical challenge.
Vetter B, Schwarz C, Kohne E, Kulozik AE	Beta-thalassaemia in the immigrant and non-immigrant German populations.	British journal of haematology 1997, 97: 266
In Germany homozygous beta-thalassaemia mainly occurs in the immigrant population from endemic regions. In non-immigrants beta-thalassaemia is rare. Heterozygous beta-thalassaemia minor, however, is more common and must be considered in the differential diagnosis of hypochromic anaemia. The clinical and molecular data of 221 homozygous patients and 256 non-immigrant German heterozygous individuals are presented. Clinically, 87% (n = 192) of the homozygotes are classified as thalassaemia major (TM) and the other 13% as thalassaemia intermedia (TI). There is a wide spectrum of 39 thalassaemia mutations which occur with relatively low frequencies in individual cases. In 17/29 TI patients 'mild' mutations have been found and in 16/29 there are mutations that are associated with increased gamma-globin gene activity. alpha-Thalassaemia is rare and found only in 3/29. In the 256 Germans with heterozygous beta-thalassaemia there are 27 different thalassaemia mutations. The three most common are Mediterranean, together accounting for 61%. Also relatively common (5%) is an otherwise rare frameshift mutation of codon 83 (FS83 deltaG). The other mutations occur in < 10 individuals. Two mutations described here are novel. One of them affects position -2 of the intron 1 splice acceptor site (IVSI-129 A-G) and the other is a deletion of a single G in codon 15/16 (FS 15/16 deltaG). These data suggest that beta-thalassaemia in Germans was introduced from the Mediterranean in about two-thirds of cases and that the remaining third has probably originated locally.
Vickers JL, Carlisle C	Choices and control: parental experiences in pediatric terminal home care.	Journal of pediatric oncology nursing 2000, 17: 12
During the past decade, palliative care at home has become an alternative option to hospital care for terminally ill children. This study describes the experience of caring for a dying child at home from a parent's perspective. A qualitative research design was used to conduct and analyze data. Nonstandardized, focused interviews were conducted with 10 families. Thematic content analysis assisted in deriving themes from the transcripts of the interviews.
Vichinsky EP	Clinical Manifestations of α-Thalassemia.	Cold Spring Harbor perspectives in medicine 2013, 3
α-Thalassemia mutations affect up to 5% of the world's population. The clinical spectrum ranges from an asymptomatic condition to a fatal in utero disease. Hemoglobin H disease results from mutations of three α-globin genes. Deletional forms result in a relatively mild anemia, whereas nondeletional mutations result in a moderate to severe disease characterized by ineffective erythropoiesis, recurrent transfusions, and growth delay. Hemosiderosis develops secondary to increased iron absorption, as well as transfusion burden. Hemoglobin Bart's hydrops fetalis is usually a fatal in utero disease caused by the absence of α genes. Population screening to identify at-risk couples is essential. Affected pregnancies result in severe fetal and maternal complications. Doppler ultrasonography with intrauterine transfusion therapy may improve the fetal prognosis but creates ethical challenges for the family and health providers.
Vieira Pinheiro J, Ahlke E, Nowak-Gottl U, Hempel G, Muller H, Lumkemann K, Schrappe M, Rath B, Fleischhack G, Mann G, Boos J	Pharmacokinetic dose adjustment of Erwinia asparaginase in protocol II of the paediatric ALL/NHL-BFM treatment protocols.	Br J Haematol 1999, 104: 313
Vieira Pinheiro P, Ahlke E, Nowak-Göttl U, Hempel G, Müller H, Lümkemann K, Schrappe M, Rath B, Fleischhack G, Mann G, Boos J	Pharmacokinetic dose adjustment of Erwinia asparaginase in protocol II of the paediatric ALL/NHL-BFM treatment protocols.	Br J Haematol 1999, 104: 313
Vieira Pinheiro J, Müller H, Schwabe D, Gunkel M, Casimiro Da Palma J, Henze G, Von Schutz V, Winkelhorst M, Wurthwein G, Boos J	Drug monitoring of low-dose PEG-asparaginase (Oncaspar TM) in children with relapsed acute lymphoblastic leukaemia.	Br J Haematol 2001, 113: 115
Viehmann S, Reinhardt D, Griesinger F, Creutzig U, Harbott J	Monitoring of minimal residual disease (MRD) in children with AML - comparison of two quantitative methods.	Monatsschrift Kinderheilkunde 2002, 150: 553
Viehmann S, Teigler-Schlegel A, Bruch J, Langebrake C, Reinhardt D, Harbott J	Monitoring of minimal residual disease (MRD) by real-time quantitative reverse transcription PCR (RQ-RT-PCR) in childhood acute myeloid leukemia with AML1/ETO rearrangement.	Leukemia 2003, 17: 1130
Virgone C, Roganovic J, Vorwerk P, Redlich A, Schneider DT, Janic D, Bien E, López-Almaraz R, Godzinski J, Osterlundh G, Stachowicz-Stencel T, Brugières L, Brecht IB, Thomas-Teinturier C, Fresneau B, Surun A, Ferrari A, Bisogno G, Orbach D	Adrenocortical tumours in children and adolescents: The EXPeRT/PARTNER diagnostic and therapeutic recommendations.	Pediatric blood & cancer 2021, 68 Suppl 4:e29025
Adrenocortical tumours (ACTs) are rare during childhood. A complete surgical resection provides the best chance of cure, but the role and efficacy of the adjuvant therapy are still controversial. Various histologic criteria of malignancy for ACTs adopted in children do not facilitate comparative studies and are not completely shared. Therefore, a sharp demarcation between benign and malignant lesions has not been recognised, making it difficult to identify who potentially needs perioperative therapy. This manuscript presents the internationally harmonised recommendations for the diagnosis and treatment of ACTs in children and adolescents, established by the European Cooperative Study Group for Paediatric Rare Tumours (EXPeRT) group within the EU-funded project PARTNER (Paediatric Rare Tumours Network - European Registry).
Vishnu P, Moreno Vanegas Y, Wadei HM, Rivera CE	Post-transplant erythrocytosis refractory to ACE inhibitors and angiotensin receptor blockers.	BMJ case reports 2018, 2018,bcr2018224622
Post-transplant erythrocytosis (PTE) is a condition with elevated haematocrit (hct) in renal allograft recipients. The mainstay of treatment is ACE inhibitors (ACEi) or angiotensin II receptor blockers (ARB), but seldom phlebotomy. PTE must be recognised early to prevent major thromboembolic events. We present a case of PTE that was refractory to blockade of renin-angiotensin system (RAS) by ACEi and ARB and required phlebotomy for control of hct. Our review of medical literature about prevalence and pathophysiology of PTE suggests that approximately 22% of patients with PTE are refractory to ACEi/ARB treatment. There are four plausible pathways that appear to play a role in causing PTE: disruption of erythropoietin regulation, mitogenic effect of the RAS on erythroid lineage, insulin-like growth factor 1 and androgenic stimulation. Presently, there is no unifying hypothesis involving these factors, but refractoriness to ACEi/ARB may represent a distinct subcategory of PTE.
Vlachos A, Ball S, Dahl N, Alter BP, Sheth S, Ramenghi U, Meerpohl J, Karlsson S, Liu JM, Leblanc T, Paley C, Kang EM, Leder EJ, Atsidaftos E, Shimamura A, Bessler M, Glader B, Lipton JM, Participants of Sixth Annual Daniella Maria Arturi International Consensus Conference	Diagnosing and treating Diamond Blackfan anaemia: results of an international clinical consensus conference.	British journal of haematology 2008, 142: 859
Diamond Blackfan anaemia (DBA) is a rare, genetically and clinically heterogeneous, inherited red cell aplasia. Classical DBA affects about seven per million live births and presents during the first year of life. However, as mutated genes have been discovered in DBA, non-classical cases with less distinct phenotypes are being described in adults as well as children. In caring for these patients it is often difficult to have a clear understanding of the treatment options and their outcomes because of the lack of complete information on the natural history of the disease. The purpose of this document is to review the criteria for diagnosis, evaluate the available treatment options, including corticosteroid and transfusion therapies and stem cell transplantation, and propose a plan for optimizing patient care. Congenital anomalies, mode of inheritance, cancer predisposition, and pregnancy in DBA are also reviewed. Evidence-based conclusions will be made when possible; however, as in many rare diseases, the data are often anecdotal and the recommendations are based upon the best judgment of experienced clinicians. The recommendations regarding the diagnosis and management described in this report are the result of deliberations and discussions at an international consensus conference.
Vlachos A, Muir E	How I treat Diamond-Blackfan anemia.	Blood 2010, 116: 3715
Diamond-Blackfan anemia (DBA) is characterized by red cell failure, the presence of congenital anomalies, and cancer predisposition. In addition to being an inherited bone marrow failure syndrome, DBA is also categorized as a ribosomopathy as, in more than 50% of cases, the syndrome appears to result from haploinsufficiency of either a small or large subunit-associated ribosomal protein. Nonetheless, the exact mechanism by which haploinsufficiency results in erythroid failure, as well as the other clinical manifestations, remains uncertain. New knowledge regarding genetic and molecular mechanisms combined with robust clinical data from several international patient registries has provided important insights into the diagnosis of DBA and may, in the future, provide new treatments as well. Diagnostic criteria have been expanded to include patients with little or no clinical findings. Patient management is therefore centered on accurate diagnosis, appropriate use of transfusions and iron chelation, corticosteroids, hematopoietic stem cell transplantation, and a coordinated multidisciplinary approach to these complex patients.
Vlachos A, Muir E	How I treat Diamond-Blackfan anemia.	Blood 2010, 116: 3715
Diamond-Blackfan anemia (DBA) is characterized by red cell failure, the presence of congenital anomalies, and cancer predisposition. In addition to being an inherited bone marrow failure syndrome, DBA is also categorized as a ribosomopathy as, in more than 50% of cases, the syndrome appears to result from haploinsufficiency of either a small or large subunit-associated ribosomal protein. Nonetheless, the exact mechanism by which haploinsufficiency results in erythroid failure, as well as the other clinical manifestations, remains uncertain. New knowledge regarding genetic and molecular mechanisms combined with robust clinical data from several international patient registries has provided important insights into the diagnosis of DBA and may, in the future, provide new treatments as well. Diagnostic criteria have been expanded to include patients with little or no clinical findings. Patient management is therefore centered on accurate diagnosis, appropriate use of transfusions and iron chelation, corticosteroids, hematopoietic stem cell transplantation, and a coordinated multidisciplinary approach to these complex patients.
De Vleeschouwer S, Fieuws S, Rutkowski S, Van Calenbergh F, Van Loon J, Goffin J, Sciot R, Wilms G, Demaerel P, Warmuth-Metz M, Soerensen N, Wolff JE, Wagner S, Kaempgen E, Van Gool SW	Postoperative adjuvant dendritic cell-based immunotherapy in patients with relapsed glioblastoma multiforme.	Clinical cancer research : an official journal of the American Association for Cancer Research 2008 May 15; 14: 3098
To investigate the therapeutic role of adjuvant vaccination with autologous mature dendritic cells (DC) loaded with tumor lysates derived from autologous, resected glioblastoma multiforme (GBM) at time of relapse.
Vogel F	Genetics of retinoblastoma.	Human genetics 1979, 52: 1
The genetic basis of retinoblastoma is reviewed and the following conclusions are drawn: 1) The mode of inheritance of the hereditary variety of retinoblastoma (R) is autosomal dominant with about 90% penetrance. 2) About 68% of inherited cases are bilateral, and about 32%, unilateral. There is an intrafamilial correlation between penetrance as measured by segregation ratio and expressivity as measured by the fraction of bilaterally affected patients. 3) The vast majority of all R patients are sporadic cases, i.e., they are the only affected members of otherwise unaffected families. The porportion of bilateral cases is much lower among sporadic than among hereditary cases. 4) All bilaterally affected patients with sporadic R and patients with unilateral sporadic R with more than one primary tumor have to be regarded as germ cell mutants; they will transmit the gene to 50% of their offspring. Only 10%-12% of unilateral sporadic cases are germ cell mutants; 88%-90% are nonhereditary; in these cases the tumor is probably caused by a somatic mutation. 5) In a minority of cases, deletion of the chromosome segment 13q14(=intersitital deletion of the long arm of chromosome 13) has been observed. In addition to R, the patients show a variable degree of general or mental retardation; often there are few external indications of a chromosome aberration. Other chromosome studies suggest anomalies of chromosome 13 in tumor tissue even in cases not showing an anomaly of this chromosome in blood cultures, and possibly a slightly increased chromosome instability. 6) Patients with bilateral, and possibly in general with hereditary, R run an increased risk of becoming affected with other tumor diseases, such as osseous sarcomas, in later life. 7) Knudson's hypothesis of two mutational steps leading to both the hereditary and the nonhereditary variants of R is discussed critically, and the alternative possibility is suggested that in the nonhereditary variant a single mutational step--possibly a small chromosome aberration--could be enough to produce a tumor. 8) Evidence indicating a possible viral origin of R is cited, and animal experiments are mentioned in which R-like tumors have successfully been produced by local DNA virus inoculation. 9) As a consequence of improved survival and reproduction of R patients, an increased in the incidence of R and in the proportion of bilateral cases among all R patients must be anticipated. 10) Detailed rules for genetic counseling in families affected by R are given.
Vogel F	Genetics of retinoblastoma.	Human genetics 1979, 52: 1
The genetic basis of retinoblastoma is reviewed and the following conclusions are drawn: 1) The mode of inheritance of the hereditary variety of retinoblastoma (R) is autosomal dominant with about 90% penetrance. 2) About 68% of inherited cases are bilateral, and about 32%, unilateral. There is an intrafamilial correlation between penetrance as measured by segregation ratio and expressivity as measured by the fraction of bilaterally affected patients. 3) The vast majority of all R patients are sporadic cases, i.e., they are the only affected members of otherwise unaffected families. The porportion of bilateral cases is much lower among sporadic than among hereditary cases. 4) All bilaterally affected patients with sporadic R and patients with unilateral sporadic R with more than one primary tumor have to be regarded as germ cell mutants; they will transmit the gene to 50% of their offspring. Only 10%-12% of unilateral sporadic cases are germ cell mutants; 88%-90% are nonhereditary; in these cases the tumor is probably caused by a somatic mutation. 5) In a minority of cases, deletion of the chromosome segment 13q14(=intersitital deletion of the long arm of chromosome 13) has been observed. In addition to R, the patients show a variable degree of general or mental retardation; often there are few external indications of a chromosome aberration. Other chromosome studies suggest anomalies of chromosome 13 in tumor tissue even in cases not showing an anomaly of this chromosome in blood cultures, and possibly a slightly increased chromosome instability. 6) Patients with bilateral, and possibly in general with hereditary, R run an increased risk of becoming affected with other tumor diseases, such as osseous sarcomas, in later life. 7) Knudson's hypothesis of two mutational steps leading to both the hereditary and the nonhereditary variants of R is discussed critically, and the alternative possibility is suggested that in the nonhereditary variant a single mutational step--possibly a small chromosome aberration--could be enough to produce a tumor. 8) Evidence indicating a possible viral origin of R is cited, and animal experiments are mentioned in which R-like tumors have successfully been produced by local DNA virus inoculation. 9) As a consequence of improved survival and reproduction of R patients, an increased in the incidence of R and in the proportion of bilateral cases among all R patients must be anticipated. 10) Detailed rules for genetic counseling in families affected by R are given.
Voges J, Sturm V, Berthold F, Pastyr O, Schlegel W, Lorenz WJ	Interstitial irradiation of cerebral gliomas in childhood by permanently implanted 125-iodine--preliminary results.	Klin Pädiatr 1990, 202: 270
Voggel S, Abele M, Seitz C, Agaimy A, Vokuhl C, Dirksen U, Bier A, Flaadt T, Classen CF, Claviez A, Schneider DT, Brecht IB	Primary lung carcinoma in children and adolescents - Clinical characteristics and outcome of 12 cases from the German registry for rare paediatric tumours (STEP).	Lung cancer 2021, 160: 66
Primary lung carcinomas are very rare paediatric tumours with an incidence of < 2/1.000.000 per year. They are clinically and histologically heterogeneous, and there are no therapeutic guidelines for this age group. Therefore, they represent a challenge for treating physicians. This analysis was performed to expand knowledge on characteristics, treatment and prognosis of primary lung carcinoma in paediatric patients.
Vokuhl C, Oyen F, Häberle B, von Schweinitz D, Schneppenheim R, Leuschner I	Small Cell Undifferentiated Hepatoblastomas (SCUD): All Malignant Rhabdoid Tumors?	Genes, chromosomes & cancer 2016,
Small cell undifferentiated hepatoblastoma (SCUD) is a rare variant of hepatoblastoma with poor outcome and loss of INI1 expression, sharing this with malignant rhabdoid tumors (MRT). We studied all tumors from the files of the Kiel Pediatric Tumor Registry (KTR) with the initial diagnosis of SCUD and MRT. After re-review, we performed immunistochemistry, FISH and MLPA for loss of expression and deletion of INI1/SMARCB1 in 23 tumors. Morphologically, 12 of the tumors had a small cell morphology, nine showed the typical picture of MRT and two were composed of both small cells and rhabdoid cells. All but one of the 23 tumors showed loss of INI1 protein expression by immunohistochemistry. Nineteen of the INI1 negative tumors were analysed by FISH technique and all showed a deletion of the INI1/SMARCB1 gene (17 homozygous deletions, 2 heterozygous deletions). We investigated 14 of these cases by MLPA and verified the deletions in all cases. In conclusion, we postulate that small cell undifferentiated hepatoblastoma (HB) is not a hepatoblastoma but represents a malignant rhabdoid tumor of the liver. This article is protected by copyright. All rights reserved.
Vokuhl C, Nourkami-Tutdibi N, Furtwängler R, Gessler M, Graf N, Leuschner I	ETV6-NTRK3 in congenital mesoblastic nephroma: A report of the SIOP/GPOH nephroblastoma study.	Pediatric blood & cancer 2018, 65
BACKGROUND: Congenital mesoblastic nephroma (MN) is a rare pediatric renal tumor representing approximately 5% of all pediatric renal tumors. Three different types of MN are distinguished histologically: classical, cellular, and mixed. A frequent genetic alteration is the translocation t(12;15) resulting in a fusion of the ETV6 gene on 12p13 and the NTRK3 gene on 15p15 that occurs almost exclusively in cellular MN. The aim of this study was to determine translocation status of a large cohort of MN with respect to tumor subtype and outcome. PROCEDURE: In total, clinical data from 111 patients were available. Sixty-seven tumors were classical MN (51%), 29 cellular MN (31%), and 15 were mixed MN (18%). From these 111 cases, 79 were analyzed by FISH and RT-PCR. RESULTS: All classical and mixed MN were translocation negative. Seventeen out of 29 (58%) cellular MN harbored the ETV6-NTRK3 translocation. Five-year relapse-free survival (RFS) and overall survival (OS) were 93.2% and 96.8% for the complete cohort. All seven relapses occurred in translocation negative tumors. Five-year RFS was significantly inferior for cellular and mixed MN compared to classic MN (89%, 80%, and 98%), whereas 5-year OS was similar (93%, 96%, and 98%). Within the group of cellular MN, patients having translocation-positive tumors had a significantly superior RFS (5-year RFS: 100% vs. 73%). CONCLUSION: The majority of cellular MNs harbor the ETV6-NTKR3 gene fusion, whereas all classic- and mixed-type MNs were translocation negative. Within the cellular subgroup, patients having translocation-positive tumors had a significantly superior RFS. © 2017 Wiley Periodicals, Inc.
Volm M, Mattern J, Stammler G, Royer-Pokora B, Schneider S, Weirich A, Ludwig R	Expression of resistance-related proteins in nephroblastoma after chemotherapy.	Int J Cancer 1995, 63: 193
von Schweinitz D, Burger D, Weinel P, Mildenberger H	Therapy of malignant liver tumors in childhood. An intermediate report of the HB-89 multicenter study of the GPOH.	Klin Pädiatr 1992, 204: 214
von Schweinitz D, Hadam M, Welte K, Mildenberger H, Pietsch T	Production of interleukin-1 beta and interleukin-6 in hepatoblastoma.	Int J Cancer 1993, 53: 728
von Schweinitz D, Burger D, Bode U, Weinel P, Erttmann R, Hecker H, Mildenberger H	Results of the HB-89 Study in treatment of malignant epithelial liver tumors in childhood and concept of a new HB-94 protocol.	Klin Pädiatr 1994, 206: 282
von Schweinitz D, Burger D, Mildenberger H	Is laparatomy the first step in treatment of childhood liver tumors?--The experience from the German Cooperative Pediatric Liver Tumor Study HB-89.	Eur J Pediatr Surg 1994, 4: 82
von Schweinitz D, Wischmeyer P, Leuschner I, Schmidt D, Wittekind C, Harms D, Mildenberger H	Clinico-pathological criteria with prognostic relevance in hepatoblastoma.	Eur J Cancer 1994, 30A: 1052
von Schweinitz D, Gluer S, Mildenberger H	Liver tumors in neonates and very young infants.	Eur J Pediatr Surg 1995, 5: 72
von Schweinitz D, Hecker H, Burger D, Mildenberger H	Surgical therapy of hepatoblastoma in childhood.	Langenbecks Arch Chir 1995, 380: 315
von Schweinitz D, Hecker H, Harms D, Bode U, Weinel P, Burger D, Erttmann R, Mildenberger H	Complete resection before development of drug resistance is essential for survival from advanced hepatoblastoma--a report from the German Cooperative Pediatric Liver Tumor Study HB-89.	J Pediatr Surg 1995, 30: 845
von Kries R, Göbel U, Hachmeister A, Kaletsch U, Michaelis J	Vitamin K and childhood cancer.	BMJ 1996, 313: 199
von Schweinitz D, Fuchs J, Mildenberger H	Surgical strategy in pediatric liver malignancies.	Langenbecks Arch Chir Suppl Kongressbd 1996, 113: 1091
von Schweinitz D, Byrd D, Hecker H, Weinel P, Bode U, Burger D, Erttmann R, Harms D, Mildenberger H	Efficiency and toxicity of ifosfamide, cisplatin and doxorubicin in the treatment of childhood hepatoblastoma. Study Committee of the Cooperative Paediatric Liver Tumour Study HB89 of the German Society for Paediatric Oncology and Haematology.	Eur J Cancer 1997, 33: 1243
von Schweinitz D, Hecker H, Schmidt-von-Arndt G, Harms D	Prognostic factors and staging systems in childhood hepatoblastoma.	Int J Cancer 1997, 74: 593
von Schweinitz D	Identification of risk groups in hepatoblastoma--another step in optimising therapy.	Eur J Cancer 2000, 36: 1343
von Schweinitz D, Faundez A, Teichmann B, Birnbaum T, Koch A, Hecker H, Gluer S, Fuchs J, Pietsch T	Hepatocyte growth-factor-scatter factor can stimulate post-operative tumor-cell proliferation in childhood hepatoblastoma.	Int J Cancer 2000, 85: 151
von Schweinitz D, Hero B, Berthold F	The impact of surgical radicality on outcome in childhood neuroblastoma.	Eur J Pediatr Surg 2002, 12: 402
von Schweinitz D, Kraus J, Albrecht S, Koch A, Fuchs J, Pietsch T	Prognostic impact of molecular genetic alterations in hepatoblastoma.	Med Pediatr Oncol 2002, 38: 104
von Schweinitz D	Neonatal liver tumours.	Semin Neonatol 2003, 8: 403
Vormoor J, Ritter J, Creutzig U, Schellong G	Inzidenz und prognostische Bedeutung von Auerstäbchen bei Kindern mit akuter myeloischer Leukämie in den Studien AML-BFM-78 and -83.	Klinische Pädiatrie 1989, 201: 227
Vormoor J, Ritter J, Creutzig U, Boos J, Heyen P, Ludwig W, Harbott J, Löffler H, Schellong G, Group for	Acute myelogenous leukaemia in children under 2 years - experiences of the West German AML Studies BFM-78-83 and -87.	British Journal of Cancer 1992, 66 (Suppl.XVIII): 63
Vormoor J, Boos J, Stahnke K, Jürgens H, Ritter J, Creutzig U	Therapy of childhood acute myelogenous leukemias.	Annals Hematology 1996, 73: 11
Vormoor J, Baersch G, Decker S, Hotfilder M, Schafer K, Pelken L, Rube C, van Valen F, Jürgens H, Dockhorn-Dworniczak B	Establishment of an In Vivo Model for Pediatric Ewing Tumors by Transplantation into NOD/scid Mice.	Pediatr Res 2001, 49: 332
Vormoor J, Klingebiel T, Jürgens H	[Current state of cord blood transplantation in childhood].	Klinische Padiatrie 2002 Jul-Aug; 214: 195
In 1989 E. Gluckman reported the successful cord blood transplantation in a boy with Fanconi anemia. Since then more than 1500 allogeneic cord blood transplantations have been performed worldwide. This has been possible because non-profit cord blood banks have been established that provide cryopreserved cord blood products from unrelated donors. However, cord blood transplantation is associated with specific risks that have sofar limited its more widespread use. Its main problem is the limited stem cell dose that is associated with a long aplasia and a high rate of engraftment failure. Therefore, cord blood is used as the stem cell source in only about 1 - 2 % of stem cell transplantations in childhood. The main advantage of cord blood transplantation lies in its low risk for graft-versus-host-disease (GvHD), one of the major causes for posttransplant morbidity and mortality particularly with unrelated stem cell donors. The low risk for GvHD is attributed to the low number of transplanted T cells and their functional immaturity. Another advantage of cord blood transplantation lies in the immediate availability of the cord blood units. Based on the experiences with allogeneic cord blood transplantation the indications for cord blood donation will be discussed.
Vora A, Andreano A, Pui CH, Hunger SP, Schrappe M, Moericke A, Biondi A, Escherich G, Silverman LB, Goulden N, Taskinen M, Pieters R, Horibe K, Devidas M, Locatelli F, Valsecchi MG	Influence of Cranial Radiotherapy on Outcome in Children With Acute Lymphoblastic Leukemia Treated With Contemporary Therapy.	Journal of clinical oncology 2016, 34: 919
We sought to determine whether cranial radiotherapy (CRT) is necessary to prevent relapse in any subgroup of children with acute lymphoblastic leukemia (ALL).
Voskaridou E, Ladis V, Kattamis A, Hassapopoulou E, Economou M, Kourakli A, Maragkos K, Kontogianni K, Lafioniatis S, Vrettou E, Koutsouka F, Papadakis A, Mihos A, Eftihiadis E, Farmaki K, Papageorgiou O, Tapaki G, Maili P, Theohari M, Drosou M, Kartasis Z, Aggelaki M, Basileiadi A, Adamopoulos I, Lafiatis I, Galanopoulos A, Xanthopoulidis G, Dimitriadou E, Mprimi A, Stamatopoulou M, Haile ED, Tsironi M, Anastasiadis A, Kalmanti M, Papadopoulou M, Panori E, Dimoxenou P, Tsirka A, Georgakopoulos D, Drandrakis P, Dionisopoulou D, Ntalamaga A, Davros I, Karagiorga M, on behalf of the Greek Haemoglobinopathies Study Group	A national registry of haemoglobinopathies in Greece: Deducted demographics, trends in mortality and affected births.	Annals of hematology 2012, epub ahead of print
Haemoglobinopathies are the most common hereditary disorders in Greece. Although there is a successful national prevention program, established 35 years ago, there is lack of an official registry and collection of epidemiological data for haemoglobinopathies. This paper reports the results of the first National Registry for Haemoglobinopathies in Greece (NRHG), recently organized by the Greek Society of Haematology. NRHG records all patients affected by thalassaemia major (TM), thalassaemia intermedia (TI),
Voth H, Oberthuer A, Simon T, Kahlert Y, Berthold F, Fischer M	Identification of DEIN, a novel gene with high expression levels in stage IVS neuroblastoma.	Molecular cancer research : MCR 2007, 5: 1276
Neuroblastoma at stage IVS, defined by dissemination to specific tissues and age <1 year at diagnosis, regularly follows spontaneous regression without cytotoxic treatment. To uncover the molecular characteristics of this subtype, Serial Analysis of Gene Expression (SAGE) profiles from stage IVS and fatal stage IV tumors were compared. A SAGE tag (GCAACTTAAC) was detected that was overrepresented in stage IVS disease and that had no reliable match in current National Center for Biotechnology Information databases of SAGE profiles, thus pointing to a novel gene. The corresponding gene, which maps to chromosome 4q33-34, was identified using a modified 3'- and 5'-rapid amplification of cDNA ends PCR and was designated as DEIN (differentially expressed in neuroblastoma). The gene comprises five transcript variants and its sequence overlaps with expressed sequences of the yet uncharacterized UniGene cluster Hs.61435. DEIN exhibits nucleotide sequence conservation over a broad range of species with an overall homology of 65% between human and mouse. As none of the predicted amino acid sequences is homologous to known proteins, it remains to be determined whether DEIN represents a coding or noncoding RNA. Northern blot analysis and semiquantitative reverse transcription-PCR showed high DEIN expression in neuroblastoma, whereas expression was absent or weak in most normal adult tissues. Analysis of 121 primary neuroblastomas by real-time reverse transcription-PCR revealed a strong association with age at diagnosis <1 year and particularly with stage IVS disease (both P < 0.001). The characteristic expression pattern of DEIN suggests a specific role of this gene in the unique biology of stage IVS tumors and may help to molecularly define this special subtype of neuroblastoma.
Voth H, Oberthuer A, Simon T, Kahlert Y, Berthold F, Fischer M	Co-regulated expression of HAND2 and DEIN by a bidirectional promoter with asymmetrical activity in neuroblastoma.	BMC molecular biology 2009, 10: 28
BACKGROUND: HAND2, a key regulator for the development of the sympathetic nervous system, is located on chromosome 4q33 in a head-to-head orientation with DEIN, a recently identified novel gene with stage specific expression in primary neuroblastoma (NB). Both genes are expressed in primary NB as well as most NB cell lines and are separated by a genomic sequence of 228 bp. The similar expression profile of both genes suggests a common transcriptional regulation mediated by a bidirectional promoter. RESULTS: Northern Blot analysis of DEIN and HAND2 in 20 primary NBs indicated concurrent expression levels of the two genes, which was confirmed by microarray analysis of 236 primary NBs (Pearson's correlation coefficient r = 0.65). While DEIN expression in the latter cohort was associated with stage 4S (p = 0.02), HAND2 expression was not associated with tumor stage. In contrast, both HAND2 and DEIN transcript levels were highly associated with age at diagnosis <12 months (p = 0.001). The intergenic region shows substantial homology in different species (89%, 72% and 53% identity between human and mouse, chicken and zebrafish, respectively) and contains many highly conserved putative transcription factor binding sites. Using luciferase reporter gene constructs, asymmetrical bidirectional promoter activity was found in four NB cell lines: In DEIN orientation, an average 3.4 fold increase in activity was observed as compared to the promoterless vector, whereas an average 15.4 fold activation was detected in HAND2 orientation. The presence of two highly conserved putative regulatory elements, one of which was shown to enhance HAND2 expression in branchial arches previously, displayed weak repressor activity for both genes. CONCLUSION: HAND2 and DEIN represent a gene pair that is tightly linked by a bidirectional promoter in an evolutionary highly conserved manner. Expression of both genes in NB is co-regulated by asymmetrical activity of this promoter and modulated by the activity of two cis-regulatory elements acting as weak repressors. The concurrent quantitative and tissue specific expression of HAND2 and DEIN suggests a functional link between both genes.
Vokuhl C, Nourkami-Tutdibi N, FurtwÃ¤ngler R, Gessler M, Graf N, Leuschner I	ETV6-NTRK3 in congenital mesoblastic nephroma: A report of the SIOP/GPOH nephroblastoma study.	Pediatric blood & cancer 2018, 65
BACKGROUND: Congenital mesoblastic nephroma (MN) is a rare pediatric renal tumor representing approximately 5% of all pediatric renal tumors. Three different types of MN are distinguished histologically: classical, cellular, and mixed. A frequent genetic alteration is the translocation t(12;15) resulting in a fusion of the ETV6 gene on 12p13 and the NTRK3 gene on 15p15 that occurs almost exclusively in cellular MN. The aim of this study was to determine translocation status of a large cohort of MN with respect to tumor subtype and outcome. PROCEDURE: In total, clinical data from 111 patients were available. Sixty-seven tumors were classical MN (51%), 29 cellular MN (31%), and 15 were mixed MN (18%). From these 111 cases, 79 were analyzed by FISH and RT-PCR. RESULTS: All classical and mixed MN were translocation negative. Seventeen out of 29 (58%) cellular MN harbored the ETV6-NTRK3 translocation. Five-year relapse-free survival (RFS) and overall survival (OS) were 93.2% and 96.8% for the complete cohort. All seven relapses occurred in translocation negative tumors. Five-year RFS was significantly inferior for cellular and mixed MN compared to classic MN (89%, 80%, and 98%), whereas 5-year OS was similar (93%, 96%, and 98%). Within the group of cellular MN, patients having translocation-positive tumors had a significantly superior RFS (5-year RFS: 100% vs. 73%). CONCLUSION: The majority of cellular MNs harbor the ETV6-NTKR3 gene fusion, whereas all classic- and mixed-type MNs were translocation negative. Within the cellular subgroup, patients having translocation-positive tumors had a significantly superior RFS. © 2017 Wiley Periodicals, Inc.
de Vries AC, Bredius RG, Lankester AC, Bierings M, Trebo M, Sedlacek P, Niemeyer CM, Zecca M, Locatelli F, van den Heuvel-Eibrink MM	HLA-identical umbilical cord blood transplantation from a sibling donor in juvenile myelomonocytic leukemia.	Haematologica 2009, 94: 302
van Schaik J, Hoving EW, Müller HL, van Santen HM	Hypothalamic-Pituitary Outcome after Treatment for Childhood Craniopharyngioma.	Frontiers of hormone research 2021, 54: 47
Although childhood-onset craniopharyngioma is a low-grade intracranial tumor with excellent prognosis in terms of overall survival, survivors may suffer from devastating consequences caused by hypothalamic damage. Disease- or treatment-related hypothalamic damage leads to disturbed hunger-satiety and thirst feelings, decreased energy expenditure, behavioral problems, disturbances of circadian rhythm, temperature dysregulation, and pituitary dysfunction. These children are at great risk for developing the metabolic syndrome and comorbidities leading to premature mortality. In this chapter, we shall discuss hypothalamic-pituitary morbidity and outcome of childhood-onset craniopharyngioma patients and future perspectives for improvement.
Vujanić GM, D'Hooghe E, Graf N, Vokuhl C, Al-Saadi R, Chowdhury T, Pritchard-Jones K, Furtwängler R	Prognostic significance of histopathological response to preoperative chemotherapy in unilateral Wilms' tumor: An analysis of 899 patients treated on the SIOP WT 2001 protocol in the UK-CCLG and GPOH studies.	International journal of cancer 2021, 149: 1332
In the SIOP Wilms' tumor (WT) studies, preoperative chemotherapy is used as primary treatment, and tumors are classified thereafter by pathologists. Completely necrotic WTs (CN-WTs) are classified as low-risk tumors. The aim of the study was to evaluate whether a subset of regressive type WTs (RT-WTs) (67%-99% chemotherapy-induced changes [CIC]) showing an exceptionally good response to preoperative chemotherapy had comparably excellent survivals as CN-WTs, and to establish a cut-off point of CIC that could define this subset. The study included 2117 patients with unilateral, nonanaplastic WTs from the UK-CCLG and GPOH-WT studies (2001-2020) treated according to the SIOP-WT-2001 protocol. There were 126 patients with CN-WTs and 773 with RT-WTs, stages I-IV. RT-WTs were subdivided into subtotally necrotic WTs (>95% CIC) (STN-WT96-99) (124 patients) and the remaining of RT-WT (RR-WT67-95) (649 patients). The 5-year event-free survival (EFS) and overall survival (OS) for CN-WTs were 95.3% (±2.1% SE) and 97.3% (±1.5% SE), and for RT-WTs 85.7% (±1.14% SE, P < .01) and 95.2% (±0.01% SE, P = .59), respectively. CN-WT and STN-WT96-99 groups showed significantly better EFS than RR-WT67-95 (P = .003 and P = .02, respectively), which remained significantly superior when adjusted for age, local stage and metastasis at diagnosis, in multivariate analysis, whereas OS were superimposable (97.3 ± 1.5% SE for CN-WT; 97.8 ± 1.5% SE for STN-WT96-99; 94.7 ± 1.0% SE for RR-WT67-95). Patients with STN-WT96-99 share the same excellent EFS and OS as patients with CN-WTs, and although this was achieved by more treatment for patients with STN-WT96-99 than for patients with CN-WT, reduction in postoperative treatment of these patients may be justified.
Vujanić GM, D'Hooghe E, Graf N, Vokuhl C, Al-Saadi R, Chowdhury T, Pritchard-Jones K, Furtwängler R	Prognostic significance of histopathological response to preoperative chemotherapy in unilateral Wilms' tumor: An analysis of 899 patients treated on the SIOP WT 2001 protocol in the UK-CCLG and GPOH studies.	International journal of cancer 2021, 149: 1332
In the SIOP Wilms' tumor (WT) studies, preoperative chemotherapy is used as primary treatment, and tumors are classified thereafter by pathologists. Completely necrotic WTs (CN-WTs) are classified as low-risk tumors. The aim of the study was to evaluate whether a subset of regressive type WTs (RT-WTs) (67%-99% chemotherapy-induced changes [CIC]) showing an exceptionally good response to preoperative chemotherapy had comparably excellent survivals as CN-WTs, and to establish a cut-off point of CIC that could define this subset. The study included 2117 patients with unilateral, nonanaplastic WTs from the UK-CCLG and GPOH-WT studies (2001-2020) treated according to the SIOP-WT-2001 protocol. There were 126 patients with CN-WTs and 773 with RT-WTs, stages I-IV. RT-WTs were subdivided into subtotally necrotic WTs (>95% CIC) (STN-WT96-99) (124 patients) and the remaining of RT-WT (RR-WT67-95) (649 patients). The 5-year event-free survival (EFS) and overall survival (OS) for CN-WTs were 95.3% (±2.1% SE) and 97.3% (±1.5% SE), and for RT-WTs 85.7% (±1.14% SE, P < .01) and 95.2% (±0.01% SE, P = .59), respectively. CN-WT and STN-WT96-99 groups showed significantly better EFS than RR-WT67-95 (P = .003 and P = .02, respectively), which remained significantly superior when adjusted for age, local stage and metastasis at diagnosis, in multivariate analysis, whereas OS were superimposable (97.3 ± 1.5% SE for CN-WT; 97.8 ± 1.5% SE for STN-WT96-99; 94.7 ± 1.0% SE for RR-WT67-95). Patients with STN-WT96-99 share the same excellent EFS and OS as patients with CN-WTs, and although this was achieved by more treatment for patients with STN-WT96-99 than for patients with CN-WT, reduction in postoperative treatment of these patients may be justified.
Vujanić GM, Graf N, D'Hooghe E, Chowdhury T, Vokuhl C, Al-Saadi R, Pritchard-Jones K, Melchior P, Furtwängler R	Outcomes of patients with Wilms' tumour stage III due to positive resection margins only: An analysis of patients treated on the SIOP-WT-2001 protocol in the UK-CCLG and GPOH studies.	International journal of cancer 2023, 152: 1640
Stage III Wilms' tumour (WT) represents a heterogeneous group which includes different criteria, but all stage III patients are treated according to the same study regiment. The aim of the study was to retrospectively analyse outcomes in patients with stage III due to positive resection margins (RM) only, sub-grouped in RM with viable (RM-v) and nonviable (RM-nv) tumour. Patients were treated pre- and postoperatively according to the SIOP-WT-2001 protocol in the UK-CCLG and GPOH WT trials and studies (2001-2020). There were 197 patients, including 134 with localised, abdominal stage III and 63 with overall stage IV, but abdominal stage III. Stage III due to RM-v had 126 patients, and due to RM-nv 71 patients. The overall 5-year local-relapse-free survival (RFS), event-free (EFS) and overall survival (OS) estimates for all patients with abdominal stage III RM were 95.7% (±SE1.5%), 85.1 (±SE2.6%) and 90.3% (±SE2.2%), respectively. Patients with stage III RM-nv had significantly better RFS and EFS than patients with RM-v (P = .027 and P = .003, respectively). A multivariate analysis showed that RM-v remained a significant factor for EFS when adjusted for age, presence of metastasis at diagnosis, histological risk group and overall stage in Cox regression analysis (P = .006). Patients with stage III due to RM-nv only exhibited no local recurrence and have a significantly better RFS and EFS than patients with RM-v. The results suggest that exclusion of RM-nv as a stage III criterion in the UMBRELLA staging system and consequent treatment reduction is warranted.
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