Dataset Information

Limited Contribution of Donor Characteristics to One-Year Survival After Hematopoietic Stem Cell Transplantation.

ABSTRACT: A large number of association studies have related donor characteristics to survival after bone marrow transplantation, for leukemia in general and specifically for acute myeloid leukemia (AML) patients. However, population-based differences often do not hold at the single transplant level. We test whether transplantation outcomes can be predicted at the single-patient level and whether such predictions can be used to better choose donors. The analysis was performed on a mixture of different diseases or with AML only, and with either patient and donor information or donor information only. We analyzed 3671 8-of-8 HLA-matched AML donor-recipient pairs and tested whether the outcome, including 1-year total and event-free survival, can be predicted from patient and donor-related factors. We used multiple machine learning and survival analysis methods. The best method is a fully connected neural network. Multiple outcomes can be predicted, with area under the specificity-sensitivity curve (AUC) values between 0.54 and 0.67 for the different outcomes. The patient age has a strong impact on prediction. However, for a given patient, when only donor or transplant information is used, limited prediction accuracy of 0.54 to 0.56 AUC for event-free survival and survival is obtained. Graft-versus-host disease and rejection after 1 year have slightly higher AUC values of around 0.59, whereas the relapse prediction accuracy was random. All donors' characteristics have a limited influence on the quality of hematopoietic stem cell transplantation for fully matched donors. Many factors with a population effect on survival have a very limited effect when combined with all other factors in a single-donor predictive model.

SUBMITTER: Israeli S

PROVIDER: S-EPMC10129847 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature

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Limited Contribution of Donor Characteristics to One-Year Survival After Hematopoietic Stem Cell Transplantation.

Israeli Sapir S Maiers Martin M Louzoun Yoram Y

Transplantation and cellular therapy 20220828 12

A large number of association studies have related donor characteristics to survival after bone marrow transplantation, for leukemia in general and specifically for acute myeloid leukemia (AML) patients. However, population-based differences often do not hold at the single transplant level. We test whether transplantation outcomes can be predicted at the single-patient level and whether such predictions can be used to better choose donors. The analysis was performed on a mixture of different dis ...[more]

PMID: 36038105

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Project description:IntroductionDonor lymphocyte infusion (DLI) is a therapeutic approach for relapse after hematopoietic stem cell transplantation (HSCT). Despite their reported efficacy, the evolution of DLI practices over time remains underexplored.MethodsThis study provided a comprehensive analysis of DLI strategies and outcomes over 30 years at a single institution. A retrospective analysis was conducted on 75 patients who underwent DLI for disease relapse between April 1994 and March 2024. The primary endpoint was the 3-year overall survival (OS) rate after DLI. Secondary endpoints included the 100-day complete remission (CR) rate and incidence of acute graft-versus-host disease (GVHD).ResultsThe median age at the first DLI was 49 years (range, 20-69 years). The most common underlying diseases in all 75 cases were acute myeloid leukemia (AML, n = 46) and myelodysplastic syndromes (MDS, n = 12). Until 2014, DLI was only performed in patients with AML (n = 14), MDS (n = 2), or chronic myeloid leukemia (n = 5). However, since 2015, patients with various diseases, including lymphoid malignancies, have also undergone DLI. Azacitidine was the most frequently used combination therapy with DLI (n = 34). Regimens including venetoclax and FLT3 inhibitors have been commonly used since 2019 (n = 18). The 3-year OS rate was 29.1% (95% CI, 18.8-40.2%). Factors negatively influencing OS included age ≥50 years and a high or very high refined disease risk index. The 100-day CR rate was 52.1%, and acute GVHD occurred in 25.3% of the patients, with no strong correlation between GVHD incidence and CR achievement. Among 18 patients who underwent three or more DLIs since 2018, 88.9% achieved remission following DLI or second HSCT, with a median follow-up of 949.5 days for survivors.ConclusionThis study highlighted the evolving trends in DLI practices and the diversification of combination therapies. Future research should focus on further validating these findings and optimizing DLI protocols to improve patient outcomes.

Project description:BackgroundAllogeneic hematopoietic stem cell transplantation (allo-HSCT) has proven to be an effective curative intervention for children with β-thalassemia major (β-TM). They are susceptible to growth disorders due various factors. The primary objective of this study was to investigate the growth and development of children with β-TM both before and after undergoing allo-HSCT during a one-year follow-up period.MethodsAccording to the international reference standards for child growth established by the World Health Organization in 2006, participants were divided into two groups based on their Z scores: weight-for-age Z scores (WAZ), height-for-age Z scores (HAZ), and body mass index-for-age Z scores (BAZ) before allo-HSCT. The first group included children with normal nutritional levels (-2≤ Z scores ≤2), while the second group consisted of children with malnutrition (Z scores <-2 or >2). Measurements of weight, height, and body mass index (BMI) were recorded at six time points: prior to allo-HSCT (T0), and 1 month (T1), 3 months (T3), 6 months (T6), 9 months (T9), and 12 months (T12) following the procedure.ResultsAmong the 56 children diagnosed with β-TM, 25% were malnourished prior to HSCT. In the group with normal nutrition, mean WAZ (P=0.04) and HAZ (P<0.001) values were significantly higher at T12 compared to T0; however, mean BAZ was similar at T12 and T0 (P=0.61). In contrast, in the malnutrition group, no significant differences were observed in mean WAZ (P=0.12), HAZ (P=0.27), or BAZ (P=0.75) values between T12 and T0. Notably, the incidence of increased HAZ was significantly more prevalent in children aged seven years or younger than in those older than seven years (P=0.01).ConclusionsChildren diagnosed with β-TM who had normal nutritional levels at the time of allo-HSCT showed significant growth improvements within one year of follow-up compared to their pre-transplantation state. Additionally, the age at which allo-HSCT was performed may substantially influence post-transplantation growth rates. Regular monitoring of growth and development from the time of diagnosis is crucial to ensure optimal nutritional status at the time of allo-HSCT, thereby enhancing the prognosis for these children after transplantation.

Dataset Information

Limited Contribution of Donor Characteristics to One-Year Survival After Hematopoietic Stem Cell Transplantation.

Publications

Limited Contribution of Donor Characteristics to One-Year Survival After Hematopoietic Stem Cell Transplantation.

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