[Outcomes of allogeneic hematopoietic stem cell transplantation for 18 patients with paroxysmal nocturnal haemoglobinuria].
Zhonghua Xue Ye Xue Za Zhi
; 36(12): 1005-10, 2015 Dec.
Article
em Zh
| MEDLINE
| ID: mdl-26759101
ABSTRACT
OBJECTIVE:
To evaluate the outcomes of allogeneic hematopoietic stem cell transplantationï¼allo-HSCTï¼for paroxysmal nocturnal haemoglobinuriaï¼PNHï¼and aplastic anemiaï¼AAï¼- PNH syndrome.METHODS:
The clinical data of 18 PNH or AA-PNH patients, including 4 classic PNH and 14 AA-PNH, received allo-HSCT from Dec 2007 to Feb 2015 were analyzed retrospectively. Nine patients received HLA-haploidentical donor HSCTï¼1 patient received salvage HLA-haploidentical donor HSCT after the graft failure of double cord blood transplantationï¼, 7 patients received HLA-identical sibling donor HSCT, and 2 HLA-identical unrelated donor HSCT. The conditioning regimens were as follow 13 patients received modified BU/CY- based regimens, 5 non- myeloablative regimens ï¼»fludarabine ï¼Fluï¼ + antithymocyte globulinï¼ATGï¼+ cyclophosphamideï¼CYï¼or busulfanï¼BUï¼ï¼½. Prophylaxis for graft- versushost diseaseï¼GVHDï¼ the patients with HLA-identical sibling donor received cyclosporineï¼CsAï¼plus short-term methotrexateï¼MTXï¼, the patients with HLA -haploidentical donor or HLA-identical unrelated donor received CsA or tacrolimusï¼FK506ï¼+ mycophenolate mofetilï¼MMFï¼+ short- term methotrexate ï¼MTXï¼.RESULTS:
All patients were engrafted successfullyï¼1 patient engrafted by haploidentical donor after the graft failure of double cord blood transplantationï¼. The median days of neutrophilsï¼ANCï¼above 0.5 × 109/L and platelets ï¼PLTï¼ more than 20 × 109/L were 11ï¼10- 26ï¼days and 15ï¼11- 120ï¼days, respectively. Three patientsï¼17.6%ï¼developed acute GVHDï¼aGVHDï¼, 2 for grade â ¡ aGVHD, 1 for grade â £ aGVHD. Of 16 patients, 2 occurred limited chronic GVHDï¼cGVHDï¼. After a median follow-up of 14.6ï¼2.0-86.7ï¼months, 3 patientsï¼17.6%ï¼died, out of which one died of severe aGVHD, one died of severe pulmonary infection, one pulmonary infection with transplant- associated thrombotic microangiopathy. The 5- year estimated disease free survival wasï¼80.5 ± 10.2ï¼%. No patient relapsed.CONCLUSION:
Allo-HSCT is an effective and curable therapy for PNH or AA-PNH with improved prognosis, and offers a valid therapeutic option for these patients before humanized monoclonal antibody against C5 are widely used clinically.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Células-Tronco Hematopoéticas
/
Hemoglobinúria Paroxística
/
Anemia Aplástica
Tipo de estudo:
Observational_studies
/
Risk_factors_studies
Limite:
Humans
Idioma:
Zh
Ano de publicação:
2015
Tipo de documento:
Article