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Klin Padiatr 2015; 227(03): 157-165
DOI: 10.1055/s-0035-1548841
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© Georg Thieme Verlag KG Stuttgart · New York

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
1   Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
,
W. Ebell
2   Pediatric Hematology/Oncology and Stem Cell Transplantation, Charité University Medicine Berlin, Berlin, Germany
,
P. Bader
3   Zentrum für Kinder- und Jugendmedizin, Klinikum der Johann-Wolfgang-Goethe-Universität, Klinik III, Frankfurt, Germany
,
R. Beier
1   Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
,
B. Burkhardt
4   Pediatric Hematology/Oncology, University of Muenster, Muenster, Germany
,
T. Feuchtinger
5   Pediatric hematology/Oncology, University Children’s Hospital Tuebingen, Tuebingen, Germany
,
R. Handgretinger
5   Pediatric hematology/Oncology, University Children’s Hospital Tuebingen, Tuebingen, Germany
,
H. Hanenberg
6   Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA
7   Department of Otorhinolaryngology and Head/Neck Surgery, Heinrich Heine University, Düsseldorf, Germany
,
U. Koehl
8   Institute of Cellular Therapeutics, Hannover Medical School, GMP-DU, IFB-Tx, Hannover, Germany
,
C. Kratz
1   Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
,
B. Kremens
9   Paediatric Hematology and Oncology, Medical Center, University of Essen, Essen, Germany
,
P. Lang
5   Pediatric hematology/Oncology, University Children’s Hospital Tuebingen, Tuebingen, Germany
,
R. Meisel
10   Department of Pediatric Hematology Oncology, University of Düsseldorf Medical School, Düsseldorf, Germany
,
I. Mueller
11   Pediatric Hematologie/Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
,
C. Roessig
4   Pediatric Hematology/Oncology, University of Muenster, Muenster, Germany
,
M. Sauer
1   Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
,
P. G. Schlegel
12   Childrens Hospitals, University of Würzburg, Germany
,
A. Schulz
13   Univ.-Klinik für Kinder- und Jugendmedizin, Ulm, Germany
,
B. Strahm
14   Pediatric Hematology and Oncology, University Freiburg, Freiburg, Germany
,
F. Thol
15   Hematology/Onkology, Hannover Medical School, Hannover, Germany
,
K. W. Sykora
1   Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
› Author Affiliations
Further Information

Publication History

Publication Date:
18 May 2015 (online)

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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
 

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