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Fludarabine‐based reduced‐intensity conditioning regimen for hematopoietic stem cell transplantation in primary hemophagocytic lymphohistiocytosis
Author(s) -
Hamidieh Amir Ali,
Pourpak Zahra,
Hashemi Susan,
Yari Kolsoum,
Fazlollahi Mohammad Reza,
Movahedi Masoud,
Behfar Maryam,
Moin Mostafa,
Ghavamzadeh Ardeshir
Publication year - 2014
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.12244
Subject(s) - medicine , cytopenia , fludarabine , hematopoietic stem cell transplantation , hemophagocytic lymphohistiocytosis , transplantation , primary immunodeficiency , regimen , melphalan , surgery , gastroenterology , chemotherapy , disease , cyclophosphamide , bone marrow
Objective Primary hemophagocytic lymphohistiocytosis ( HLH ) is a life‐threatening condition that clinically characterized by fever, hepatosplenomegaly, and cytopenia. Hematopoietic stem cell transplantation ( HSCT ) is the only curative treatment option for patients diagnosed with primary HLH . Methods In this prospective study, we analyzed the outcome of 10 pediatric patients with primary HLH who had received HSCT , using reduced‐intensity conditioning ( RIC ) regimen from 2007 to 2012. The median age at transplantation was 22.6 months (range: 6–60). All of the patients received the same RIC regimen based on the use of fludarabine in combination with melphalan and horse antithymocyte globulin ( ATG ). Cyclosporine and methylprednisolone were used as graft‐vs.‐host disease ( G v HD ) prophylaxis. Results Hematopoietic engraftment occurred in all patients. At the present time, 8 patients with a median follow‐up of 39 months are still alive and all of them are disease free. Acute and chronic Gv HD developed in 6 and 2 patients, retrospectively. Two patients died of sepsis and chronic Gv HD during the study. Conclusion Because of pretransplant infections caused by underlying immunodeficiency in patients with primary HLH , the use of less toxic regimen with RIC seems to be highly effective in this regard. Recipients of RIC transplant, with either full or mixed chimerism, had a long‐term survival rate with no manifestation of primary HLH symptoms.