Consensus of German Transplant Centers on Hematopoietic Stem Cell Transplantation in Fanconi Anemia

M. M. Chao; W. Ebell; P. Bader; R. Beier; B. Burkhardt; T. Feuchtinger; R. Handgretinger; H. Hanenberg; U. Koehl; C. Kratz; B. Kremens; P. Lang; R. Meisel; I. Mueller; C. Roessig; M. Sauer; P. G. Schlegel; A. Schulz; B. Strahm; F. Thol; K. W. Sykora

doi:10.1055/s-0035-1548841

Klin Padiatr 2015; 227(03): 157-165
DOI: 10.1055/s-0035-1548841

Report

Consensus of German Transplant Centers on Hematopoietic Stem Cell Transplantation in Fanconi Anemia

Konsensus Empfehlungen Deutscher Transplantationszentren zur hämatopoetischen Stammzelltransplantation bei Fanconi Anämie

M. M. Chao

¹Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany

,

W. Ebell

²Pediatric Hematology/Oncology and Stem Cell Transplantation, Charité University Medicine Berlin, Berlin, Germany

,

P. Bader

³Zentrum für Kinder- und Jugendmedizin, Klinikum der Johann-Wolfgang-Goethe-Universität, Klinik III, Frankfurt, Germany

,

R. Beier

¹Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany

,

B. Burkhardt

⁴Pediatric Hematology/Oncology, University of Muenster, Muenster, Germany

,

T. Feuchtinger

⁵Pediatric hematology/Oncology, University Children’s Hospital Tuebingen, Tuebingen, Germany

,

R. Handgretinger

⁵Pediatric hematology/Oncology, University Children’s Hospital Tuebingen, Tuebingen, Germany

,

H. Hanenberg

⁶Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA

⁷Department of Otorhinolaryngology and Head/Neck Surgery, Heinrich Heine University, Düsseldorf, Germany

,

U. Koehl

⁸Institute of Cellular Therapeutics, Hannover Medical School, GMP-DU, IFB-Tx, Hannover, Germany

,

C. Kratz

¹Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany

,

B. Kremens

⁹Paediatric Hematology and Oncology, Medical Center, University of Essen, Essen, Germany

,

P. Lang

⁵Pediatric hematology/Oncology, University Children’s Hospital Tuebingen, Tuebingen, Germany

,

R. Meisel

¹⁰Department of Pediatric Hematology Oncology, University of Düsseldorf Medical School, Düsseldorf, Germany

,

I. Mueller

¹¹Pediatric Hematologie/Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

,

C. Roessig

⁴Pediatric Hematology/Oncology, University of Muenster, Muenster, Germany

,

M. Sauer

¹Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany

,

P. G. Schlegel

¹²Childrens Hospitals, University of Würzburg, Germany

,

A. Schulz

¹³Univ.-Klinik für Kinder- und Jugendmedizin, Ulm, Germany

,

B. Strahm

¹⁴Pediatric Hematology and Oncology, University Freiburg, Freiburg, Germany

,

F. Thol

¹⁵Hematology/Onkology, Hannover Medical School, Hannover, Germany

,

K. W. Sykora

¹Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany

› Institutsangaben

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative therapy for the severe hematopoietic complications associated with Fanconi anemia (FA). In Germany, it is estimated that 10–15 transplants are performed annually for FA. However, because FA is a DNA repair disorder, standard conditioning regimens confer a high risk of excessive regimen-related toxicities and mortality, and reduced intensity regimens are linked with graft failure in some FA patients. Moreover, development of graft-versus-host disease is a major contributing factor for secondary solid tumors. The relative rarity of the disorder limits HSCT experience at any single center. Consensus meetings were convened to develop a national approach for HSCT in FA. This manuscript outlines current experience and knowledge about HSCT in FA and, based on this analysis, general recommendations reached at these meetings.

Zusammenfassung

Die allogene Stammzelltransplantation (HSCT) ist zurzeit die einzige kurative Therapie der schweren hämatopoetischen Komplikationen der Fanconi-Anämie (FA). In Deutschland werden etwa 10–15 Transplantationen pro Jahr bei Patienten mit FA durchgeführt. Da die FA auf einem Defekt der DNA-Reparatur beruht, können Standard-Konditionierungsprotokolle zu hoher Toxizität und Mortalität führen, während Regime mit reduzierter Intensität ein Risiko für Transplantatversagen in sich tragen. Hinzu kommt, dass die Entwicklung der Transplantat-gegen-Empfänger-Erkrankung ein wichtiger Risikofaktor für die Entwicklung von sekundären soliden Tumoren ist. Die relative Seltenheit der Erkrankung führt dazu, dass die Erfahrung jedes einzelnen Transplantationszentrums mit der Erkrankung begrenzt ist. Konsensus-Treffen mit dem Ziel der Entwicklung eines bundesweiten Vorgehens bei der Transplantation der FA wurden abgehalten. Dieses Manuskript beschreibt die langjährige Erfahrung und den aktuellen Kenntnisstand zur Transplantation bei FA sowie den darauf basierenden Konsens, der auf diesen Treffen erzielt wurde.

Key words

fanconi anemia - stem cell transplantation - consensus - recommendations

Schlüsselwörter

Fanconi Anämie - Stammzelltransplantation - Konsensus - Empfehlungen

Volltext

Referenzen

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