Comparison of Late Effects and Quality of Life by Donor Type in Long-Term Survivors of Severe Aplastic Anemia after Hematopoietic Cell Transplantation

With improved outcomes of patients who have undergone allogeneic hematopoietic cell transplantation (HCT) for severe aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (SAA), there is a growing population of survivors. Current literature lacks details regarding relevant late effects in these survivors. It is also important to compare late effects and quality of life (QOL) by donor type given the increasing use of alternative donors for HCT in SAA. Integral to Fred Hutchinson Cancer Center's long-term follow-up, HCT recipients are sent annual surveys designed to understand patients' health status and QOL. We analyzed surveys of patients who underwent HCT for SAA and responded to a survey between 2015 and 2023. Self-reported educational/vocational status, chronic health conditions, medications, and QOL data were captured. QOL was measured using Short-Form 36 (SF-36) questionnaires; scores were normalized to the general population mean of 50 with a standard deviation of 10 points. Multivariable logistic regression was performed to study the associations between donor type and chronic health condition (heart, pulmonary, and renal, musculoskeletal, sexual dysfunction, cataracts) and medication (cardiopulmonary, metabolic/skeletal, endocrine, gastrointestinal, emotional health) categories. Multivariable linear regression was used to study the associations between donor type and SF-36 domain and summary scores. The analysis included 122 survivors who underwent HCT between 1972 and 2021. The median (range) ages at HCT and survey were 20.8 (0.8 to 67.3) and 51.2 (5.6 to 78.9) yr, respectively. Median interval between HCT and survey was 26.0 (1.1 to 47.1) yr. Most respondents underwent matched related donor matched related donor: A bone marrow/stem cell donor that is a sibling or another family relation to the patient. (MRD) HCT (n = 82, 67%), matched unrelated donor unrelated donor: A donor that is not a sibling or other familial relation of the patient (recipient). (MUD) HCT being next most common (n = 24, 20%), followed by alternative donor HCT (n = 16, 13%). Among the 86 patients of employment eligible age, 42 (49%) survivors were working full-time and 21 (24%) part-time at the time of the survey. The most common reported chronic health conditions were problems with sexual desire, erection, ejaculation, vaginal dryness, or pain (35%), cataracts (20%), and reduced bone mineral density (20%). The median number of problems requiring medications was 1 (interquartile range 0 to 3); most were used for hypertension (34%), gastroesophageal reflux (22%), and high cholesterol (21%). Compared to survivors after MRD HCT, those after alternative donor HCT (OR 9.9, 95% confidence interval 1.8, 54.1) were more likely to report chronic health conditions affecting the musculoskeletal system. Among 96 respondents who provided SF-36 data, mean (SD) physical component summary score of 49.5 (11.0) and the mental component summary score of 49.9 (12.3) were comparable to general population norms. Compared to MRD, survivors after MUD HCT were noted to have a better score for bodily pain (parameter estimate 9.2, 95% confidence interval 1.4, 17.0). No significant associations between donor types and SF-36 scores were noted in any of the other domains. Patients undergoing HCT for SAA remain at risk for late effects emphasizing the need for lifelong monitoring. However, it is encouraging to find that QOL among long-term survivors was similar to that of the general population. We did not detect statistically significant differences in late effects or QOL by donor type, although comparisons were limited by sample size and survivor/respondent bias.