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 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.
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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G
Dufort
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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L
Castillo
Transfusion Medicine Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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S
Pisano
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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M
Castiglioni
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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P
Carolina
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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I
Andrea
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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E
Simon
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
S
S
Zuccolo
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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M
Schelotto
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
F
F
Morosini
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
I
I
Pereira
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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P
Amarillo
Statistics Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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A
Silveira
Radiotherapy Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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L
Guerrero
Radiotherapy Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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V
Ferreira
Transfusion Medicine Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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A
Tiscornia
Transfusion Medicine Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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R
Mezzano
Transfusion Medicine Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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F
Lemos
Transfusion Medicine Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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B
Boggia
Radiotherapy Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
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A
Quarnetti
Transfusion Medicine Department, Asociación Española, Montevideo, Uruguay.
J
J
Decaro
Pediatric Hemato-Oncology Department, Centro Hospitalario Pereira Rosell, Montevideo, Uruguay.
A
A
Dabezies
D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000517:prevention & control*; D019337:Hematologic Neoplasms / Q000401:mortality / Q000628:therapy*; D006801:Humans; D015994:Incidence; D008212:Lymphocyte Depletion; D008297:Male; D012008:Recurrence; D016019:Survival Analysis; D000075442:Transplantation, Haploidentical / Q000009:adverse effects / Q000379:methods* / Q000401:mortality / Q000592:standards; D016896:Treatment Outcome; D014583:Uruguay
D003520:Cyclophosphamide