Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study.
Patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor need new therapeutic approaches. Hematopoietic SCT (HSCT) using mismatched or haploidentical related donors has been used in the past, but was associated with a significant risk of GVHD and mortality. Recently, the use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GVHD in recipients of haploidentical HSCT, but the majority of reports have focused on patients with hematology malignancies. We describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. Stem cell sources were BM (N=13) or PBSCs (N=3). The rate of neutrophil engraftment was 94% and of platelet engraftment was 75%. Two patients had secondary graft failure and were successfully salvaged with another transplant. Three patients developed acute GVHD being grades 2-4 in two. Five patients have died and the 1-year OS was 67.1% (95% confidence interval: 36.5-86.4%). In our small series, the use of a reduced-intensity conditioning with post-transplant Cy in haploidentical BMT was associated with high rates of engraftment and low risk of GVHD in patients with relapsed/refractory SAA.
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
I
I
Esteves
Hospital das Clínicas do Parana, Curitiba, Brazil.
C
C
Bonfim
Hospital das Clínicas do Parana, Curitiba, Brazil.
R
R
Pasquini
Hospital das Clínicas do Parana, Curitiba, Brazil.
V
V
Funke
Hospital das Clínicas do Parana, Curitiba, Brazil.
N F
NF
Pereira
Hospital Sírio Libanês, São Paulo, Brazil.
V
V
Rocha
Hospital Sírio Libanês, São Paulo, Brazil.
Y
Y
Novis
Hospital Sírio Libanês, São Paulo, Brazil.
C
C
Arrais
Hospital Amaral Carvalho, Jau, Brazil.
V
V
Colturato
Hospital Amaral Carvalho, Jau, Brazil.
M P
MP
de Souza
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
M
M
Torres
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
J F
JF
Fernandes
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
F R
FR
Kerbauy
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
A A F
AA
Ribeiro
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
F P S
FP
Santos
Centro de Oncologia e Hematologia Familia Dayan-Daycoval, Hospital Israelita Albert Einstein, São Paulo, Brazil.
N
N
Hamerschlak
D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control*; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D019172:Transplantation Conditioning
D007166:Immunosuppressive Agents; D003520:Cyclophosphamide