The role of donor type and pre‐transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia
Background The goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA). Methods This retrospective, multi‐center study included 52 SA...
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| Published in | Pediatric transplantation Vol. 28; no. 4; pp. e14784 - n/a |
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| Main Authors | , , , , , , , , , , , , , , , |
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| Abstract | Background
The goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).
Methods
This retrospective, multi‐center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first‐ or second‐line treatment.
Results
The median age at HSCT for all 52 patients was 15 years (range 1–25). The 3‐year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4–99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5–99] for haploidentical (N = 13), and 71% [95% CI 36–99] for matched unrelated donors (MUD) (N = 7). The 3‐year OS was 81% [95% CI 69.7‐99] for all patients, 90.5% [95% CI 79.5‐99] for non‐IST patients (N = 27), and 70% [95% CI 51–99] for IST patients (N = 24) (log‐rank p = .04). Survival of haploidentical HSCT (haplo‐HSCT) recipients with post‐transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non‐IST patients (N = 3) and 80% for IST patients (N = 10). The 3‐year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3–99] for haplo‐HSCT (N = 10) and 66.7% [95% CI 28.7–99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log‐rank was not significant(p = .65).
Conclusions
Patients receiving MUD and haplo‐HSCT with PTCy had similar outcomes, suggesting that haplo‐HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT. |
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| AbstractList | The goal of this study was to assess the effect of donor type and pre-transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).BACKGROUNDThe goal of this study was to assess the effect of donor type and pre-transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).This retrospective, multi-center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first- or second-line treatment.METHODSThis retrospective, multi-center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first- or second-line treatment.The median age at HSCT for all 52 patients was 15 years (range 1-25). The 3-year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4-99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5-99] for haploidentical (N = 13), and 71% [95% CI 36-99] for matched unrelated donors (MUD) (N = 7). The 3-year OS was 81% [95% CI 69.7-99] for all patients, 90.5% [95% CI 79.5-99] for non-IST patients (N = 27), and 70% [95% CI 51-99] for IST patients (N = 24) (log-rank p = .04). Survival of haploidentical HSCT (haplo-HSCT) recipients with post-transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non-IST patients (N = 3) and 80% for IST patients (N = 10). The 3-year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3-99] for haplo-HSCT (N = 10) and 66.7% [95% CI 28.7-99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log-rank was not significant(p = .65).RESULTSThe median age at HSCT for all 52 patients was 15 years (range 1-25). The 3-year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4-99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5-99] for haploidentical (N = 13), and 71% [95% CI 36-99] for matched unrelated donors (MUD) (N = 7). The 3-year OS was 81% [95% CI 69.7-99] for all patients, 90.5% [95% CI 79.5-99] for non-IST patients (N = 27), and 70% [95% CI 51-99] for IST patients (N = 24) (log-rank p = .04). Survival of haploidentical HSCT (haplo-HSCT) recipients with post-transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non-IST patients (N = 3) and 80% for IST patients (N = 10). The 3-year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3-99] for haplo-HSCT (N = 10) and 66.7% [95% CI 28.7-99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log-rank was not significant(p = .65).Patients receiving MUD and haplo-HSCT with PTCy had similar outcomes, suggesting that haplo-HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT.CONCLUSIONSPatients receiving MUD and haplo-HSCT with PTCy had similar outcomes, suggesting that haplo-HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT. Background The goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA). Methods This retrospective, multi‐center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first‐ or second‐line treatment. Results The median age at HSCT for all 52 patients was 15 years (range 1–25). The 3‐year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4–99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5–99] for haploidentical (N = 13), and 71% [95% CI 36–99] for matched unrelated donors (MUD) (N = 7). The 3‐year OS was 81% [95% CI 69.7‐99] for all patients, 90.5% [95% CI 79.5‐99] for non‐IST patients (N = 27), and 70% [95% CI 51–99] for IST patients (N = 24) (log‐rank p = .04). Survival of haploidentical HSCT (haplo‐HSCT) recipients with post‐transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non‐IST patients (N = 3) and 80% for IST patients (N = 10). The 3‐year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3–99] for haplo‐HSCT (N = 10) and 66.7% [95% CI 28.7–99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log‐rank was not significant(p = .65). Conclusions Patients receiving MUD and haplo‐HSCT with PTCy had similar outcomes, suggesting that haplo‐HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT. BackgroundThe goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).MethodsThis retrospective, multi‐center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first‐ or second‐line treatment.ResultsThe median age at HSCT for all 52 patients was 15 years (range 1–25). The 3‐year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4–99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5–99] for haploidentical (N = 13), and 71% [95% CI 36–99] for matched unrelated donors (MUD) (N = 7). The 3‐year OS was 81% [95% CI 69.7‐99] for all patients, 90.5% [95% CI 79.5‐99] for non‐IST patients (N = 27), and 70% [95% CI 51–99] for IST patients (N = 24) (log‐rank p = .04). Survival of haploidentical HSCT (haplo‐HSCT) recipients with post‐transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non‐IST patients (N = 3) and 80% for IST patients (N = 10). The 3‐year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3–99] for haplo‐HSCT (N = 10) and 66.7% [95% CI 28.7–99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log‐rank was not significant(p = .65).ConclusionsPatients receiving MUD and haplo‐HSCT with PTCy had similar outcomes, suggesting that haplo‐HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT. The goal of this study was to assess the effect of donor type and pre-transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA). This retrospective, multi-center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first- or second-line treatment. The median age at HSCT for all 52 patients was 15 years (range 1-25). The 3-year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4-99] for matched related donors (MRD) (N = 24), 84% [95% CI 63.5-99] for haploidentical (N = 13), and 71% [95% CI 36-99] for matched unrelated donors (MUD) (N = 7). The 3-year OS was 81% [95% CI 69.7-99] for all patients, 90.5% [95% CI 79.5-99] for non-IST patients (N = 27), and 70% [95% CI 51-99] for IST patients (N = 24) (log-rank p = .04). Survival of haploidentical HSCT (haplo-HSCT) recipients with post-transplant cyclophosphamide (PTCy) (N = 13) was excellent for both groups: 100% for non-IST patients (N = 3) and 80% for IST patients (N = 10). The 3-year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3-99] for haplo-HSCT (N = 10) and 66.7% [95% CI 28.7-99] for MUD (N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log-rank was not significant(p = .65). Patients receiving MUD and haplo-HSCT with PTCy had similar outcomes, suggesting that haplo-HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT. Abstract Background The goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA). Methods This retrospective, multi‐center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first‐ or second‐line treatment. Results The median age at HSCT for all 52 patients was 15 years (range 1–25). The 3‐year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4–99] for matched related donors (MRD) ( N = 24), 84% [95% CI 63.5–99] for haploidentical ( N = 13), and 71% [95% CI 36–99] for matched unrelated donors (MUD) ( N = 7). The 3‐year OS was 81% [95% CI 69.7‐99] for all patients, 90.5% [95% CI 79.5‐99] for non‐IST patients ( N = 27), and 70% [95% CI 51–99] for IST patients ( N = 24) (log‐rank p = .04). Survival of haploidentical HSCT (haplo‐HSCT) recipients with post‐transplant cyclophosphamide (PTCy) ( N = 13) was excellent for both groups: 100% for non‐IST patients ( N = 3) and 80% for IST patients ( N = 10). The 3‐year OS for patients with previous IST by donor type in groups where >5 patients were available was 78.8% [95% CI 52.3–99] for haplo‐HSCT ( N = 10) and 66.7% [95% CI 28.7–99] for MUD ( N = 6). Although it appears that patients receiving HSCT ≥6 months after the start of IST had worse survival, the number of patients in each category was small and log‐rank was not significant( p = .65). Conclusions Patients receiving MUD and haplo‐HSCT with PTCy had similar outcomes, suggesting that haplo‐HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT. |
| Author | Galvez, Jorge Horn, Biljana Kashif, Reema Cline, Jessica Chellapandian, Deepak McNerney, Kevin Crawford, David Alperstein, Warren Castillo, Paul Ligon, John Milner, Jordan Ziga, Edward Joyce, Michael Dalal, Mansi Lee, Jin‐Ju Fort, John |
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| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/38766976$$D View this record in MEDLINE/PubMed |
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contributor: fullname: Klein OR |
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The goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell... The goal of this study was to assess the effect of donor type and pre-transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation... Abstract Background The goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell... BackgroundThe goal of this study was to assess the effect of donor type and pre‐transplant immunotherapy (IST) on outcomes of hematopoietic stem cell... |
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| SubjectTerms | Adolescent Adult Anemia Anemia, Aplastic - therapy Aplastic anemia bone marrow transplant Child Child, Preschool Clinical trials Cyclophosphamide Donors Female haploidentical Hematopoietic Stem Cell Transplantation Hematopoietic stem cells Humans Immunosuppression Immunosuppression Therapy - methods Immunosuppressive Agents - therapeutic use Immunotherapy Infant Male Patients pediatric Pediatrics Retrospective Studies severe aplastic anemia Stem cell transplantation Tissue Donors Treatment Outcome Young Adult Young adults |
| Title | The role of donor type and pre‐transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia |
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