Haploidentical hematopoietic stem cell transplantation in children with high-risk hematologic malignancies: outcomes with two different strategies for GvHD prevention. Ex vivo T-cell depletion and post-transplant cyclophosphamide: 10 years of experience at a single center

Forty patients with high-risk hematologic malignancies, median age 9 years, underwent haploidentical-HSCT from April 2005 to April 2015. Seventeen patients were transplanted with CD3-depleted PBSCs by negative selection (TCD group) following a reduced-intensity conditioning regimen (RIC), and 23 pat...

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Bibliographic Details
Published in:	Bone marrow transplantation (Basingstoke) Vol. 51; no. 10; pp. 1354 - 1360
Main Authors:	Dufort, G, Castillo, L, Pisano, S, Castiglioni, M, Carolina, P, Andrea, I, Simon, E, Zuccolo, S, Schelotto, M, Morosini, F, Pereira, I, Amarillo, P, Silveira, A, Guerrero, L, Ferreira, V, Tiscornia, A, Mezzano, R, Lemos, F, Boggia, B, Quarnetti, A, Decaro, J, Dabezies, A
Format:	Journal Article
Language:	English
Published:	London Nature Publishing Group UK 01-10-2016 Nature Publishing Group
Subjects:	13 631/250/1904 692/699/1541/1990/1673 692/699/1541/1990/283/1897 692/699/67/1990/283/2125 Blood cancer Bone marrow Care and treatment CD3 antigen Cell Biology Child Clonal selection Conditioning Cyclophosphamide Cyclophosphamide - therapeutic use Depletion Diagnosis Dosage and administration Female Graft Survival Graft vs Host Disease - prevention & control Graft vs. host disease Graft-versus-host reaction Health aspects Hematologic Neoplasms - mortality Hematologic Neoplasms - therapy Hematology Hematopoietic stem cell transplantation Hematopoietic stem cells Humans Incidence Internal Medicine Leukocytes (neutrophilic) Lymphocyte Depletion Lymphocytes T Male Medicine Medicine & Public Health Negative selection original-article Platelets Prevention Public Health Recurrence Risk Stem cell transplantation Stem Cells Survival Survival Analysis T cells Transplantation Transplantation, Haploidentical - adverse effects Transplantation, Haploidentical - methods Transplantation, Haploidentical - mortality Transplantation, Haploidentical - standards Transplants & implants Treatment Outcome Uruguay
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Summary:	Forty patients with high-risk hematologic malignancies, median age 9 years, underwent haploidentical-HSCT from April 2005 to April 2015. Seventeen patients were transplanted with CD3-depleted PBSCs by negative selection (TCD group) following a reduced-intensity conditioning regimen (RIC), and 23 patients received T-cell-replete PBSCs followed by post-transplantation cyclophosphamide (PT-Cy group) after myeloablative conditioning ( n =16) or RIC ( n =7). Outcomes are reported for the TCD and PT-Cy recipients, respectively. Engraftment was achieved in 88% versus 100%. Median time to neutrophils>500/μL was 10 days versus 15 days. Platelets>20 000/μL occurred at a median of 16 days versus 20 days, respectively. Transplant-related mortality (TRM) was 24% versus 26% at 1 year. The cumulative incidence (CI) of grade III−IV acute GvHD was 7% versus 5%, and chronic GvHD 9% versus 53% ( P =0.029). Relapse at 2 years was 31% versus 24%. Actuarial overall survival rates at 2 years were 47% versus 48%. Causes of death were infections ( n =3), sinusoidal obstructive syndrome ( n =4), acute GvHD ( n =2) and relapse ( n =9). These results indicate that haploidentical-HSCT is feasible in Uruguay. The TRM rate is of concern and should be the focus of continuing attention. Chronic GvHD risk was higher in the PT-Cy approach, so modifications are justified.
Bibliography:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	0268-3369 1476-5365
DOI:	10.1038/bmt.2016.161