Bone Marrow Transplant Recipients During Childhood at Increased Risk for Severe Chronic Health Conditions

Patients who underwent allogeneic bone marrow transplantation (BMT) as children were at increased risk for severe or life-threatening chronic health conditions. These findings were presented during the American Society of Hematology (ASH) 62nd Annual Meeting and Exposition.

For this study, 848 patients who survived at least 2 years after a BMT during early adulthood or adolescence (aged 22 years or younger) and their siblings or parents (n=515) were recruited. Participants were assessed by questionnaire for sociodemographic characteristics and health status.

At the time of study, 563 of the BMT recipients were alive and 285 were deceased after surviving at least 2 years.

At the time of transplant, patients were median 11.5 years old (range, 0.4 to 22.0). Patients received their transplant for acute lymphocytic leukemia (29%), acute myeloid leukemia/myelodysplastic syndrome (28%), severe aplastic anemia (13%), or other reasons (30%). The majority of donors were related to the BMT recipient (54%) and most tissue was sourced from bone marrow (75%).

Incidence of grades 3 to 4 health conditions by aged 30 years was significantly elevated among BMT survivors (38.5%±2.7%) compared with their siblings or parents (5.4%±1.0%; P <.0001). The odds of developing a severe health condition was 8.9-fold higher (95% CI, 6.4-12.5) among BMT recipients.

BMT survivors were at increased risk for developing cataracts (odds ratio [OR], 48.2; 95% CI, 17.9-129.5), heart disease (OR, 11.4; 95% CI, 3.9-33.3), diabetes (OR, 11.1; 95% CI,3.5-34.8), thyroid nodules (OR, 6.6; 95% CI, 2.6-17.0), joint replacements (OR, 4.4; 95% CI, 1.7-10.9), and sensorineural disorders (OR, 3.2; 95% CI, 1.5-6.8) by age 30 years.

Incidence of grades 3 to 5 health conditions by age 40 years was 60.5%±3.0% among BMT survivors. The risk for severe conditions was increased among patients exposed to total body irradiation (hazard ratio [HR], 1.7; 95% CI, 1.2-2.3), patients who received peripheral blood stem cell tissue (HR, 1.51; 95% CI, 1.06-2.15; P =.02), were older than 12 years at time of transplant (HR, 1.4; 95% CI, 1.1-1.8), and women (HR, 1.3; 95% CI, 1.0-1.6).

This study was limited by its small sample size and may have been biased by excluding patients who died within 2 years of their transplant.

Anna Holmqvist, MD, PhD, coauthor of the study, concluded, “By the age of 35, almost half of the BMT recipients had developed a severe, life threatening, or fatal condition. The findings of the present study provide evidence for long-term anticipatory risk for survivors of BMT performed in childhood, from the time of transplant continuing throughout life.”

Disclosure: Multiple authors declared affiliations with industry. Please refer to the original abstract for a full list of disclosures.

Reference

Holmqvist AS, Chen Y, Wu J, et al. Severe/life-threatening/fatal chronic health conditions (CHCs) after allogenic blood or marrow transplantation (BMT) in childhood — a report from the BMT Survivor Study (BMTSS). Presented at: American Society of Hematology (ASH) 62nd Annual Meeting and Exposition; December 5-8, 2020. Abstr 69.