HSCT for beta-thalassemia improved long-term quality of life

Patients with beta-thalassemia who underwent hematopoietic stem cell transplantation experienced similar long-term quality of life as individuals in the general population, according to study results.

Giorgio La Nasa, MD, of the Bone Marrow Transplant Center at R. Binaghi Hospital in Italy, and colleagues conducted the investigation to assess health-related quality of life among individuals more than 20 years after hematopoietic stem cell transplantation (HSCT) for beta-thalassemia.

The analysis included 109 individuals who underwent transplantation in the 1980s and 1990s. Eligible participants completed the Medical Outcomes Study 36-Item Short-Form Health Survey and the Functional Assessment of Cancer Therapy–Bone Marrow Transplant.

Median age at transplantation was 12 years (range, 1-36 years). The median age at the time of survey completion was 34 years (range, 21-48), for a median follow-up duration of 22.8 years (range, 11.7-30.3).

La Nasa and colleagues performed separate comparisons with adjustments for patient age at transplantation, as well as the presence or absence of graft-versus-host disease (GVHD). The investigators also controlled for sociodemographic and clinical variables.

Results suggested those who underwent transplantation experienced similar long-term health-related quality of life as those in the general population.

Researchers observed differences only with regard to the general health scale (-8.9; 95% CI, -15.0 to -2.7). Those who underwent transplantation had comparable mental health, education level, employment status, marital status, living arrangements and birth rate.

Researchers determined older age at transplantation, as well as the development of GVHD, were associated with poorer long-term quality of life. Patients who developed GVHD after transplantation demonstrated lower general health scores than those who did not (-17.8; 95% CI, -27.4 to -8.1).

La Nasa and colleagues conducted an additional analysis of health-related quality of life among a cohort of 124 age- and sex-matched patients who received conventional therapy for beta-thalassemia. The researchers determined those who underwent conventional therapy demonstrated poorer outcomes than those who underwent transplantation.

“Although our findings provide clear evidence of good health-related quality of life and return to normal lifestyle in long-term ex-thalassemia patients, the choice of undergoing HSCT remains difficult,” La Nasa and colleagues wrote. “Over the past few years, the availability of oral iron chelators and increasingly safe and efficient transfusion therapy has radically improved the life expectancy of thalassemia patients. On the other hand, HSCT offers patients the possibility of a definitive cure with a relatively low risk of transplantation-related mortality. The availability of novel data on health-related quality of life in the long term should provide both physicians and patients alike with a better comprehension of the advantages and potential risks of HSCT and assist them in the treatment decision-making process.”

Perspective

Adetola Kassim

  • Hematopoietic stem cell transplantation (HSCT) offers a curative option for patients with beta-thalassemia, however long-term data on health-related quality of life (HRQoL) are lacking.

    This cross-sectional study compares HRQoL outcomes of patients who had undergone HSCT between July 1982 and May 1998 with previously published Italian population norms, as well as 124 thalassemia patients conventionally treated with blood transfusions and iron chelation therapy. All patients received myeloablative conditioning from HLA-identical sibling donor.

    After a median follow-up of 22.8 years (range, 11.7-30.3), 74.3% still reported presence of at least one comorbidity (primarily metabolic, endocrine and infertility issues). Long-term HRQoL measures of transplanted patients were similar to that of the general population, but they were better than nontransplanted patients. The development of graft-versus-host disease (GVHD) and older age at HSCT had a negative impact on HRQoL outcomes. GVHD had a negative impact on the incidence of comorbidities.

    Limitations include lack of data on baseline HRQoL, transplant-related mortality and geographic limitations. Improved transfusion practices, availability of effective oral iron chelators and advances in HSCT technology make the long-term outlook brighter for thalassemia patients. Prospective long-term data on HRQoL on various therapeutic options should assist both physicians and patients in making better-informed decisions.

    • Adetola Kassim, MS, MBBS
    • Associate professor of Medicine
      Vanderbilt-Ingram Cancer Center
  • Disclosures: Kassim reports n relevant financial disclosures.