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Letermovir for Cytomegalovirus infection in pediatric patients undergoing allogenic hematopoietic stem cell transplantation: a real-life study by the Infectious Diseases Working Group of Italian Association of Pediatric Hematology-Oncology (AIEOP)
Galaverna, F., Baccelli, F., Zama, D., Tridello, G., Masetti, R., Soncini, E., Mura, R., Barzaghi, F., Colombini, A., Prunotto, G., et al
Bone marrow transplantation. 2024
Abstract
Letermovir prophylaxis revolutionized the approach to Cytomegalovirus infection in adult hematopoietic stem cell transplant (HCT), while data in pediatric setting are still lacking. We retrospectively analyzed 87 HCT children transplanted in 11 AIEOP centers receiving letermovir as off-label indication between January 2020 and November 2022. Letermovir was used as primary, secondary prophylaxis or CMV treatment in 39, 26 and 22 cases, respectively; no discontinuation due to toxicity was reported. Median duration was 100 days (14-256) for primary and 96 days (8-271) for secondary prophylaxis, respectively. None of the patients experienced CMV-clinically significant reactivation during Letermovir primary prophylaxis; one patient developed breakthrough infection during secondary prophylaxis, and 10 and 1 patient experienced asymptomatic CMV-reactivation and CMV-primary infection after drug discontinuation, respectively. Median duration of letermovir in CMV treatment was 40 days (7-134), with 4/22 patients suffering CMV-pneumonia, with an overall response rate of 86.4%. With a median follow-up of 10.7 months (8.2-11.8), estimated 1-year overall survival was 86%; no CMV-related deaths were reported in prophylaxis groups. This is the largest report on Letermovir use in pediatric HCT; real-life data confirm an excellent toxicity profile, with high efficacy as CMV prophylaxis; results in CMV-infection treatment should be investigated in larger, prospective trials.
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Long-term complications after allogeneic hematopoietic stem cell transplantation for pediatric patients with acute leukemia or myelodysplastic syndrome given either a Treosulfan- or a Busulfan-based conditioning regimen: results of an AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica) retrospective study
Saglio, F., Pagliara, D., Zecca, M., Balduzzi, A., Cattoni, A., Prete, A., Tambaro, F. P., Faraci, M., Calore, E., Locatelli, F., et al
Transplantation and cellular therapy. 2024
Abstract
BACKGROUND Patients given hematopoietic stem cell transplantation (HSCT) during their childhood for hematological malignancies have an increased risk of developing long-term sequelae that are in part attributable to the conditioning regimen. OBJECTIVE The present study aimed to assess the occurrence of long-term toxicities in a population of children undergone to HSCT for hematological malignancies using either Treosulfan or Busulfan in the conditioning regimen. STUDY DESIGN Cumulative Incidence of growth impairment, alteration of gonadal function, alteration of the thyroid function, cataract, incidence of secondary malignant neoplasia and alteration of pulmonary function were retrospectively evaluated by univariable and multivariable analysis in a population of 521 pediatric patients affected by acute leukemias and myelodysplastic syndromes treated in 20 Italian Transplant Centers affiliated to AIEOP (Associazione Italiana Ematologia ed Oncologia Pediatrica) RESULTS The median duration of the follow up of the entire study population was of 7,1 years (range 1 -16 years). Overall, patients given Busulfan developed long-term toxicities in a larger proportion of cases compared to patients treated with Treosulfan (34% versus 20% p=0.01). In univariable analysis, patients having received Treosulfan developed gonadal toxicity in 10% (95%CI: 3-15) of the cases compared with 38% (95%CI: 24-39) of Bu-treated patients (p=0,02) and this finding was confirmed by multivariable analysis (Relative Risk: 0,51 95%CI: 0,34-0,76 p=0.0009). For all the other long-term toxicities our study did not show a statistically significant association between their occurrence and the use of either Busulfan or Treosulfan. CONCLUSIONS This study provides evidence that the use of Treosulfan is correlated with a reduced incidence of gonadal toxicity in children undergoing HSCT for hematological malignancies.
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Cytogenetic abnormalities predict survival after allogeneic hematopoietic stem cell transplantation for pediatric acute myeloid leukemia: a PDWP/EBMT study
Sharma, A., Galimard, J. E., Pryce, A., Bhoopalan, S. V., Dalissier, A., Dalle, J. H., Locatelli, F., Jubert, C., Mirci-Danicar, O., Kitra-Roussou, V., et al
Bone marrow transplantation. 2024
Abstract
Poor-risk (PR) cytogenetic/molecular abnormalities generally direct pediatric patients with acute myeloid leukemia (AML) to allogeneic hematopoietic stem cell transplant (HSCT). We assessed the predictive value of cytogenetic risk classification at diagnosis with respect to post-HSCT outcomes in pediatric patients. Patients younger than 18 years at the time of their first allogeneic HSCT for AML in CR1 between 2005 and 2022 who were reported to the European Society for Blood and Marrow Transplantation registry were subgrouped into four categories. Of the 845 pediatric patients included in this study, 36% had an 11q23 abnormality, 24% had monosomy 7/del7q or monosomy 5/del5q, 24% had a complex or monosomal karyotype, and 16% had other PR cytogenetic abnormalities. In a multivariable model, 11q23 (hazard ratio [HR] = 0.66, P = 0.03) and other PR cytogenetic abnormalities (HR = 0.55, P = 0.02) were associated with significantly better overall survival when compared with monosomy 7/del7q or monosomy 5/del5q. Patients with other PR cytogenetic abnormalities had a lower risk of disease relapse after HSCT (HR = 0.49, P = 0.01) and, hence, better leukemia-free survival (HR = 0.55, P = 0.01). Therefore, we conclude that PR cytogenetic abnormalities at diagnosis predict overall survival after HSCT for AML in pediatric patients.
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Defibrotide plus best standard of care compared with best standard of care alone for the prevention of sinusoidal obstruction syndrome (HARMONY): a randomised, multicentre, phase 3 trial
Grupp, S. A., Corbacioglu, S., Kang, H. J., Teshima, T., Khaw, S. L., Locatelli, F., Maertens, J., Stelljes, M., Stepensky, P., Lopez, P., et al
The Lancet. Haematology. 2023
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Editor's Choice
Abstract
BACKGROUND Sinusoidal obstruction syndrome, also known as veno-occlusive disease, is a potentially life-threatening complication of haematopoietic stem-cell transplantation (HSCT). We aimed to compare defibrotide prophylaxis plus best supportive care versus best supportive care alone for sinusoidal obstruction syndrome prevention after HSCT. METHODS This open-label, randomised, multicentre, phase 3 trial was done in 104 centres in 14 countries. Patients who were at least 1 month old, were scheduled to receive allogeneic HSCT (adult [aged >16 years] or paediatric [aged >1 month to ≤16 years] patients) or autologous HSCT (paediatric patients only), and were at high risk or very high risk of developing sinusoidal obstruction syndrome were eligible for inclusion. Patients were randomly assigned (1:1) by an interactive web response system to receive intravenous defibrotide 25 mg/kg per day (four equal doses [6·25 mg/kg per dose]) and best supportive care (determined by individual institutional guidelines; defibrotide prophylaxis group) or best supportive care only (best supportive care group). Randomisation was stratified by sinusoidal obstruction syndrome risk, age, and country. The primary endpoint, sinusoidal obstruction syndrome-free survival at day 30 after HSCT, was assessed by an independent Endpoint Adjudication Committee in the intention-to-treat (ITT) population. Safety was assessed in all patients who received protocol treatment. The trial is registered with ClinicalTrials.gov, NCT02851407. FINDINGS Between Jan 11, 2017, and Oct 20, 2020, 372 patients (172 [46%] women and 200 [54%] men; median age 14·0 years [IQR 4·0-41·0] were randomly assigned to the defibrotide prophylaxis group (n=190) or best supportive care group (n=182; ITT population). On the basis of recommendations from the Independent Data Monitoring Committee following completion of the planned interim analysis in the first 280 recruited patients on April 29, 2020, enrolment was prematurely stopped for presumed futility. At the final analysis, sinusoidal obstruction syndrome-free survival by day 30 after HSCT was 67% (95% CI 58-74) in the defibrotide prophylaxis group and 73% (62-80) in the best supportive care group (HR 1·27 [95% CI 0·84-1·93]; p=0·85). Treatment-emergent adverse events were similar between groups during the randomised prophylaxis phase; most treatment-emergent adverse events were related to the transplantation rather than to study drug. The most common grade 3 or 4 treatment-emergent adverse events were stomatitis (grade 3, 52 [29%] of 181 patients in the defibrotide prophylaxis group and 56 [32%] of 174 patients in the best supportive care group; grade 4, two [1%] in the defibrotide prophylaxis group and two [1%] in the best supportive care group) and febrile neutropaenia (grade 3, 51 [28%] in the defibrotide prophylaxis group and 52 [30%] in the best supportive care group; grade 4, no patients in the defibrotide prophylaxis group and three [2%] in the best supportive care group). Serious treatment-emergent adverse events occurred in 74 (41%) of 181 patients in the defibrotide prophylaxis group and 61 (35%) of 174 patients in the best supportive care group. In the rescue phase, when patients in both treatment groups received defibrotide as rescue treatment, fatal treatment-related adverse events occurred in one (4%) of 25 patients in the defibrotide prophylaxis group (intracranial haemorrhage) and one (3%) of 31 patients in the best supportive care group (sinusoidal obstruction syndrome). INTERPRETATION Defibrotide did not show a benefit in the prophylaxis of sinusoidal obstruction syndrome. Additional studies of carefully selected patients at high risk of sinusoidal obstruction syndrome after HSCT are warranted. FUNDING Jazz Pharmaceuticals.
PICO Summary
Population
Adults and children at least one month old who were scheduled to receive allogeneic transplant or children 1 month – 16 years scheduled to receive autologous transplant, and at high or very high risk of developing sinusoidal obstruction syndrome (n=372)
Intervention
Defibrotide prophylaxis plus best supportive care (n=190)
Comparison
Best supportive care alone (n=182)
Outcome
On the basis of recommendations from the Independent Data Monitoring Committee following completion of the planned interim analysis in the first 280 recruited patients on April 29, 2020, enrolment was prematurely stopped for presumed futility. At the final analysis, sinusoidal obstruction syndrome-free survival by day 30 after HSCT was 67% (95% CI 58-74) in the defibrotide prophylaxis group and 73% (62-80) in the best supportive care group (HR 1·27 [95% CI 0·84-1·93]; p=0·85). Treatment-emergent adverse events were similar between groups during the randomised prophylaxis phase; most treatment-emergent adverse events were related to the transplantation rather than to study drug. Serious treatment-emergent adverse events occurred in 74 (41%) of 181 patients in the defibrotide prophylaxis group and 61 (35%) of 174 patients in the best supportive care group. In the rescue phase, when patients in both treatment groups received defibrotide as rescue treatment, fatal treatment-related adverse events occurred in one (4%) of 25 patients in the defibrotide prophylaxis group (intracranial haemorrhage) and one (3%) of 31 patients in the best supportive care group (sinusoidal obstruction syndrome).
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Treosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease: a randomized phase 2 trial
Sykora, K. W., Beier, R., Schulz, A., Cesaro, S., Greil, J., Gozdzik, J., Sedlacek, P., Bader, P., Schulte, J., Zecca, M., et al
Bone marrow transplantation. 2023
Abstract
Optimal conditioning prior to allogeneic hematopoietic stem cell transplantation for children with non-malignant diseases is subject of ongoing research. This prospective, randomized, phase 2 trial compared safety and efficacy of busulfan with treosulfan based preparative regimens. Children with non-malignant diseases received fludarabine and either intravenous (IV) busulfan (4.8 to 3.2 mg/kg/day) or IV treosulfan (10, 12, or 14 g/m(2)/day). Thiotepa administration (2 × 5 mg/kg) was at the investigator's discretion. Primary endpoint was freedom from transplantation (treatment)-related mortality (freedom from TRM), defined as death between Days -7 and +100. Overall, 101 patients (busulfan 50, treosulfan 51) with at least 12 months follow-up were analyzed. Freedom from TRM was 90.0% (95% CI: 78.2%, 96.7%) after busulfan and 100.0% (95% CI: 93.0%, 100.0%) after treosulfan. Secondary outcomes (transplantation-related mortality [12.0% versus 3.9%]) and overall survival (88.0% versus 96.1%) favored treosulfan. Graft failure was more common after treosulfan (n = 11), than after busulfan (n = 2) while all patients were rescued by second procedures except one busulfan patient. CTCAE Grade III adverse events were similar in both groups. This study confirmed treosulfan to be an excellent alternative to busulfan and can be safely used for conditioning treatment in children with non-malignant disease.
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Gut microbiota diversity before allogeneic hematopoietic stem cell transplantation as a predictor of mortality in children
Masetti, R., Leardini, D., Muratore, E., Fabbrini, M., D'Amico, F., Zama, D., Baccelli, F., Gottardi, F., Belotti, T., Ussowicz, M., et al
Blood. 2023;142(16):1387-1398
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Editor's Choice
Abstract
The correlation existing between gut microbiota diversity and survival after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has so far been studied in adults. Pediatric studies question whether this association applies to children as well. Stool samples from a multicenter cohort of 90 pediatric allo-HSCT recipients were analyzed using 16S ribosomal RNA amplicon sequencing to profile the gut microbiota and estimate diversity with the Shannon index. A global-to-local networking approach was used to characterize the ecological structure of the gut microbiota. Patients were stratified into higher- and lower-diversity groups at 2 time points: before transplantation and at neutrophil engraftment. The higher-diversity group before transplantation exhibited a higher probability of overall survival (88.9% ± 5.7% standard error [SE] vs 62.7% ± 8.2% SE; P = .011) and lower incidence of grade 2 to 4 and grade 3 to 4 acute graft-versus-host disease (aGVHD). No significant difference in relapse-free survival was observed between the 2 groups (80.0% ± 6.0% SE vs 55.4% ± 10.8% SE; P = .091). The higher-diversity group was characterized by higher relative abundances of potentially health-related microbial families, such as Ruminococcaceae and Oscillospiraceae. In contrast, the lower-diversity group showed an overabundance of Enterococcaceae and Enterobacteriaceae. Network analysis detected short-chain fatty acid producers, such as Blautia, Faecalibacterium, Roseburia, and Bacteroides, as keystones in the higher-diversity group. Enterococcus, Escherichia-Shigella, and Enterobacter were instead the keystones detected in the lower-diversity group. These results indicate that gut microbiota diversity and composition before transplantation correlate with survival and with the likelihood of developing aGVHD.
PICO Summary
Population
Children receiving allogeneic HSCT at multiple centres in Italy and Poland (n=90)
Intervention
Analysis of gut microbiota using 16S ribosomal RNA amplicon to estimate diversity with the Shannon index
Comparison
Patients were stratified into higher- and lower- diversity groups at 2 time points: before transplantation and at neutrophil engraftment (n=45 for each group)
Outcome
The higher-diversity group before transplantation exhibited a higher probability of overall survival (88.9% ± 5.7% standard error [SE] vs 62.7% ± 8.2% SE) and lower incidence of grade 2 to 4 and grade 3 to 4 acute graft-versus-host disease (aGVHD). No significant difference in relapse-free survival was observed between the 2 groups (80.0% ± 6.0% SE vs 55.4% ± 10.8% SE). The higher-diversity group was characterized by higher relative abundances of potentially health-related microbial families, such as Ruminococcaceae and Oscillospiraceae. In contrast, the lower-diversity group showed an overabundance of Enterococcaceae and Enterobacteriaceae. Network analysis detected short-chain fatty acid producers, such as Blautia, Faecalibacterium, Roseburia, and Bacteroides, as keystones in the higher-diversity group. Enterococcus, Escherichia-Shigella, and Enterobacter were instead the keystones detected in the lower-diversity group.
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Low rate of nonrelapse mortality in under 4-year-olds with ALL given chemo-conditioning for HSCT: Phase III FORUM study
Bader, P., Poetschger, U., Dalle, J. H., Moser, L. M., Balduzzi, A. C., Ansari, M., Buechner, J., Güngör, T., Ifversen, M., Kriván, G., et al
Blood advances. 2023
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). In young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of two TBI-free conditioning regimens in children with ALL <4 years old. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children were transplanted and observed for ≥6 months (median follow-up: 3 years). 3-year OS was 0.63 (95% confidence interval [95% CI]: 0.52-0.72) and 0.76 (95% CI: 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (p = 0.075), respectively. 3-year EFS was 0.52 (95% CI: 0.41-0.61) and 0.51 (95% CI: 0.39-0.62), respectively (p = 0.794). Cumulative incidence of non-relapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI: 0.02-0.12) versus 0.03 (95% CI: <0.01-0.09) (p = 0.406) and 0.42 (95% CI: 0.31-0.52) versus 0.45 (95% CI: 0.34-0.56) (p = 0.920), respectively. Grade >1 acute graft-versus-host disease (GvHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% receiving Flu/Thio/Treo (p = 0.049), while grade 3-4 occurred in 10% and 9% (p = 0.813). 3-year incidence of chronic GvHD was 0.07 (95% CI: 0.03-0.13) versus 0.05 (95% CI: 0.02-0.11), respectively (p = 0.518). In conclusion, both chemo-conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure.
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Busulfan-fludarabine- or treosulfan-fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties
Cseh, A., Galimard, J. E., de la Fuente, J., Isgro, A., Zecca, M., Garwer, B., Biffi, A., Aljurf, M., Sundin, M., Belendez, C., et al
British journal of haematology. 2023
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Editor's Choice
Abstract
How important is choice of conditioning regimen in allogeneic haematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD)? We compared HSCT outcomes by conditioning regimen in paediatric patients with SCD from the EBMT registry. In 2010-2020, 251 patients aged <18 years underwent a first matched sibling donor (MSD) HSCT with conditioning based on busulfan-fludarabine (bu-flu; n = 89) or treosulfan-fludarabine (treo-flu; n = 162). In the bu-flu and treo-flu groups, 51.7% and 99.4% of patients, respectively, received thiotepa. Median follow-up was 2.7 years. Two-year overall survival (OS) was 98.7% (95% confidence interval [CI]: 90.9-99.8) with bu-flu and 99.3% (95% CI: 95.2-99.9) with treo-flu (p = 0.63). Grade III-IV acute graft-versus-host disease (GVHD) at 100 days was 2.4% (95% CI: 0.4-7.5) and 0.6% (0.1%-3.2%) for bu-flu and treo-flu respectively (p = 0.25). The 2-year incidence of extensive chronic GVHD was 1.5% (95% CI: 0.1-7.3) with bu-flu and 8.0% (95% CI: 4.1-13.3) with treo-flu (p = 0.057). These multinational data confirm the excellent curative capacity of MSD HSCT with myeloablative conditioning. Both conditioning regimens yielded excellent OS, low rates of acute and chronic GVHD, and low rates of graft failure.
PICO Summary
Population
Children who underwent transplant for sickle cell disease, identified from the EBMT registry (n=251)
Intervention
Conditioning based on busulfan-fludarabine (bu-flu, n=89)
Comparison
Conditoning based on treosulfan-fludarabine (treo-flu, n=162).
Outcome
Median follow-up was 2.7 years. Two-year overall survival (OS) was 98.7% (95% confidence interval [CI]: 90.9-99.8) with bu-flu and 99.3% (95% CI: 95.2-99.9) with treo-flu. Grade III-IV acute graft-versus-host disease (GVHD) at 100 days was 2.4% (95% CI: 0.4-7.5) and 0.6% (0.1%-3.2%) for bu-flu and treo-flu respectively. The 2-year incidence of extensive chronic GVHD was 1.5% (95% CI: 0.1-7.3) with bu-flu and 8.0% (95% CI: 4.1-13.3) with treo-flu.
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TCRαβ/CD19 cell-depleted HLA-haploidentical transplantation to treat pediatric acute leukemia: updated final analysis
Merli, P., Algeri, M., Galaverna, F., Bertaina, V., Lucarelli, B., Boccieri, E., Becilli, M., Quagliarella, F., Rosignoli, C., Biagini, S., et al
Blood. 2023
Abstract
TCRαβ/CD19-cell depletion is a promising graft manipulation technique frequently used in the context of HLA-haploidentical hematopoietic stem cell transplantation (HSCT). We previously reported the results of a phase I-II clinical trial (NCT01810120) to assess the safety and the efficacy of this type of ex-vivo T cell-depletion in 80 children with acute leukemia, showing promising survival outcomes. We now report an updated analysis on a cohort of 213 children with a longer follow-up (median value of 47.6 months for surviving patients). With a 5-year cumulative incidence of non-relapse mortality of 5.2% (95% confidence interval, CI, 2.8-8.8) and a cumulative incidence of relapse of 22.7% (95% CI, 16.9-29.2), projected 10-year overall and disease-free survival (DFS) were 75.4% (95% CI 68.6-80.9) and 71.6% (95% CI 64.4-77.6), respectively. Cumulative incidence of both grade II-IV acute and chronic GvHD were low (14.7% and 8.1%, respectively). In a multivariable analysis for DFS including also type of disease, use of total body irradiation in the conditioning regimen [hazard ratio (HR) 0.5 (95% CI, 0.26-0.98, p=0.04)], disease status at HSCT [CR>3 versus CR1/2; HR 2.23 (95% CI, 1.20-4.16, p=0.01] and high levels of pre-HSCT minimal residual disease [HR 2.09 (95% CI, 1.01-4.33, p=0.04)] were independently associated with outcome. In summary, besides confirming the good outcome results already reported (which are almost superimposable to those of transplant from HLA-matched donors), this clinical update allows the identification of patients at higher risk of treatment failure for whom personalized approaches, aimed at reducing the risk of relapse, are warranted.
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Mother donors improve outcomes after HLA haploidentical transplantation: A Study by the Cellular Therapy and Immunobiology Working Party of the EBMT
Ruggeri, L., Eikema, D. J., Bondanza, A., Noviello, M., van Biezen, A., de Wreede, L. C., Crucitti, L., Vago, L., Ciardelli, S., Bader, P., et al
Transplantation and cellular therapy. 2022
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Editor's Choice
Abstract
BACKGROUND Trans-placental trafficking of maternal and foetal cells during pregnancy establishes long-term, reciprocal micro-chimerism in both mother and child. As a consequence, the immune system of the mother may become sensitized to paternal histocompatibility antigens. It has been hypothesized that mother's "exposure" to paternal HLA haplotype antigens during pregnancy may affect transplantation outcomes when the mother acts as donor for the child. In T-cell depleted HLA haploidentical hematopoietic transplants, maternal donors have been shown to improve transplantation outcomes (Stern et al. Blood, 2008;112: 2990-2995). OBJECTIVES AND STUDY DESIGN The present retrospective multicenter study was conducted on behalf of Cellular Therapy and Immunobiology Working Party of the EBMT. It involved 409 patients (102 pediatric and 307 adult) with acute leukemia who were given HLA-haploidentical hematopoietic transplants. The goal of the study was to evaluate the role of maternal donors in a large cohort of haploidentical transplants. RESULTS Transplants from maternal donors were associated with lower relapse incidence in T-cell depleted (HR: 2.13 (1.16-3.92), p= 0.018) as well as in a limited series of unmanipulated, in vivo T-cell depleted transplants (HR: 4.15 (0.94-18.35), P=0.06) and also better graft-vs-host disease/relapse-free survival in T-cell depleted transplants (HR: 1.67 (1.02-2.73), p = 0.04). CONCLUSION These results indicate that the mother should be the preferred donor in order to provide better graft-vs-host disease/relapse-free survival in T-cell depleted HLA-haploidentical transplants for acute leukemia.
PICO Summary
Population
Adults and children with acute leukemia who underwent HSCT from a haploidentical family donor and were reported to the EBMT registry (n=409; 102 paediatric and 307 adult)
Intervention
Maternal donor (n=96)
Comparison
Other family donor (n=313)
Outcome
Transplants from maternal donors were associated with lower relapse incidence in T-cell depleted (HR: 2.13 (1.16-3.92) as well as in a limited series of unmanipulated, in vivo T-cell depleted transplants (HR: 4.15 (0.94-18.35) and also better graft-vs-host disease/relapse-free survival in T-cell depleted transplants (HR: 1.67 (1.02-2.73).