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ctt-journal > Golubovskaya et al. (Abstract)

Golubovskaya et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 12
doi: 10.3205/ctt-2011-No12-abstract30

© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported

Abstract accepted for "5th Raisa Gorbacheva Memorial Meeting Hematopoietic Stem Cell Transplantation in Children and Adults", Saint Petersburg, Russia, September 18–20, 2011

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Hematopoietic stem cell transplantation from alternative donors for patients with Fanconi anemia and Diamond-Blackfan anemia: a single center experience

Irina K. Golubovskaya, Alexander D. Kulagin, Natalia V. Stancheva, Elena V. Semenova, Sergey N. Bondarenko, Alexander L. Alyanskiy, Elena V. Babenko, Ludmila S. Zubarovskaya, Boris V. Afanasyev

R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, St. Petersburg Pavlov State Medical University, St. Petersburg, Russia

Correspondence: Irina K. Golubovskaya, R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, St. Petersburg Pavlov State Medical University, 6/8, L. Tolstoy str., St. Petersburg, 197022, Russia, E-mail:golub@rambler.ru

Abstract

Aim: To analyze the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) from alternative donors on Fanconi anemia (FA) and Diamond-Blackfan anemia (DBA).

Methods: Between February 2007 and June 2011 six patients with FA and three patients with DBA underwent alternative donor allo-HSCT. The median patient age was 9.5 years (range 3.7–18.5). Eight patients received allo-HSCT from matched unrelated donors. Haploidentical donor allo-HSCT was performed in one patient with FA-associated acute myeloid leukemia. A non-myeloablative fludarabine-containing conditioning regimen was used for all patients. All patients were transfusion-dependent at the time of HSCT.

Results: Complete donor engraftment was achieved in 6 patients. One patient with DBA had initial autologous hematopoietic recovery, which was followed by a second transplant that resulted in 50% donor chimerism. Acute and chronic GVHD occurred in 5 and 2 patients respectively. Six patients are alive and well with a median follow-up of 11 (range 1–29) months after transplant. The causes of death of 3 patients were hemorrhage, acute GVHD grade IV (gut, liver), and transplant-related toxicity.

Conclusions: Our small study confirms encouraging results of alternative donor allo-HSCT for patients with FA and steroid-resistant transfusion-dependent DBA.

Keywords: Fanconi anemia, Diamond-Blackfan anemia, allogeneic hematopoietic stem cell transplantation, alternative donor