Stem cell transplant may improve quality of life for SCD patients
Post-transplant scores for life quality are similar to general population: Study
In the years after undergoing a stem cell transplant, people with sickle cell disease (SCD) exhibit quality-of-life scores that are similar to norms for the general population, according to a recent analysis.
“In addition to the well-documented survival benefits of [stem cell transplant] for SCD, this study describes the potential psychosocial benefit,” researchers wrote.
Findings were detailed in a letter to the editor, titled “Long-term quality of life after hematopoietic cell transplant for sickle cell disease in childhood: A STELLAR interim analysis,” published in the American Journal of Hematology.
A stem cell transplant, more formally known as a hematopoietic cell transplant or HCT, is a procedure to replace a patient’s blood stem cells with those from a healthy donor, usually a sibling. The transplanted stem cells can then give rise to new healthy red blood cells that do not carry an SCD-causing mutation.
Successful stem cell transplant can cure sickle cell, improve patient survival
When successful, HCT can effectively cure sickle cell and improve patient survival. Yet, the effects of HCT on quality of life, particularly in the long term, are less clear.
To know more, scientists conducted an analysis of data from a registry that’s tracking the long-term clinical outcomes of SCD patients undergoing HCT. The registry is called STELLAR, short for Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry.
“Given the debilitating nature of SCD, studying the impact of treatment on HrQoL [health-related quality of life] is critical,” the researchers wrote. “When considering HCT as curative therapy, it is important to understand HrQoL in the context of standard-of-care SCD management and the general population.”
The analysis included data on 27 people who completed quality-of-life assessments when they were children. They were a median age of 7.4 years at the time of HCT and 10.9 years when they completed the surveys.
There also was data from 16 patients who completed the surveys as adults. They were a median age of 14.5 years at the time of HCT and 22.9 years when they completed the surveys.
Slightly different instruments were used to assess quality of life in the two age groups.
Patients exhibited quality-of-life scores within normal range
Results showed, for most aspects of quality of life, patients exhibited scores that were within the normal bounds for the general population.
“Our interim analysis demonstrated that long-term HrQoL following HCT is largely within population norms,” the researchers wrote. Since other studies have consistently suggested that life quality in SCD patients tends to be poorer than in the general population, these findings imply that HCT improves life quality for patients, though the researchers noted the study was limited by its lack of data on life-quality assessments performed before the transplant.
Among children, life-quality scores specifically related to mobility were slightly worse than what’s typically seen in the overall population. In adults, scores related to social participation were also slightly worse than normative scores.
Statistical analyses also suggested, among adults, an older age at the time of HCT was associated with significantly better social participation scores, but worse physical function scores. This finding “suggests that age at HCT may also have a significant impact on HCT-associated physical morbidity that must be considered,” the researchers wrote.
Analyses of adults also indicated male patients tended to report less fatigue after HCT than their female counterparts.
“The potential role of biological sex on this outcome must be studied further,” the researchers wrote, noting a need to take into account other factors, such as sleep quality and the effects of sex-related hormones.
STELLAR is continuing to collect data on SCD patients undergoing HCT. The researchers noted that further data from this registry may help shed additional light on how a stem cell transplant may affect the life of SCD patients.
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