People who undergo hematopoietic cell transplant (HCT) for severe aplastic anemia (SAA) need lifelong monitoring, but their QOL is as good as the general population’s, results of a new survey-based study suggest.
“I was pleasantly surprised to see that patients who received matched related donor transplant had similar quality of life when compared to those who received matched unrelated or alternative (haploidentical, cord blood, or unrelated person) transplant,” says lead study author Neel S. Bhatt, MBBS, MPH, whose poster was presented February 22 at the 2024 Tandem Meetings of the American Society for Transplantation and Cellular Therap and Center for International Blood & Marrow Transplant Research.
“We found no differences in late effects and quality of life between donor types, but this could be due to the long interval between the transplant and the survey completion,” he adds.
People who have had HCT for SAA live longer, but details about the late effects they experience are absent from the literature. “Most of the literature on long-term complications after transplant comes from patients who underwent HCT for malignancies,” Dr. Bhatt says. “As the number of alternative donors for SAA increases, providers need to know how late effects and QOL may vary by donor type.”
25 Years Following HCT, Survivors Have Normal QOL
To fill that knowledge gap, Dr. Bhatt and his colleagues conducted a retrospective analysis of patients who received HCT for SAA and responded to at least one survey from the Fred Hutchinson Cancer Center long-term follow-up annual patient recovery questionnaire between 2015 and 2023. Respondents reported their educational and vocational status, chronic conditions, and medications. They also completed the QOL short-form 36 (SF-36) questionnaire. The researchers normalized the scores to the general population mean of 50 with a standard deviation of 10 points, and they used Student’s t-test to compare SF-36 scores between donor types.
The 122 participants had a median age of 20.8 years at HCT and 50.4 when they took the survey. The median time from transplant to the survey was 25.7 years.
Overall, 67% of respondents received HCT using matched related donors, 20% had matched unrelated donors, and 13% had alternative donors. Bone marrow, the most common graft source, was used in 93% of all donor types. Acute and chronic graft-versus-host disease occurred in 48% and 8% of respondents, respectively, and subsequent neoplasms occurred in 3%. Of the working-age survivors, 48% were employed full-time and 24% part-time at the time of the survey.
Participants reported various chronic conditions, including sexual dysfunction in 36.2%, cataracts in 25.4%, and osteopenia or osteoporosis in 24.5%. They had a median of 1 medical problem requiring medications), most commonly hypertension, high cholesterol, or gastroesophageal reflux. For the 111 respondents with SF-36 data, the mean physical component and mental component summary scores were 49.4 and 49.9, respectively, with no differences in scores between donor types (Table).
“These patients do have some health complications, but they are not impacting their quality of life. Therefore, when deciding treatment options for patients with aplastic anemia, transplant deserves an important consideration,” Dr. Bhatt advises.
Ongoing Assessment Advised and Research Planned
Dr. Bhatt hopes these study results encourage clinicians to routinely assess their post-transplant aplastic anemia patients for education and work status, late effects, and QOL.
“The numbers for matched unrelated and alternative donor transplants are small. But over time, as we do more of these transplants, we will capture prospective late-effect and QOL data to check if these results hold steady,” he explains. “We also hope to do similar analyses for patients who undergo blood or stem cell transplant for sickle cell disease, thalassemia, immune-related disorders, inherited bone marrow failures, and other nonmalignant diseases.”
