Endocrine Sequelae in Pediatric Hematopoietic Stem Cell Transplant
Recipients with Sickle Cell Disease: A Retrospective Cohort Analysis
Abstract
Background: The NIH non-myeloablative regimen has been successfully
implemented for pediatric sickle cell disease (SCD) patients undergoing
matched sibling donor (MSD) hematopoietic stem cell transplant (HSCT) in
an effort to prevent late complications, including infertility and other
endocrine sequelae. This retrospective cohort analysis evaluated the
prevalence of endocrine complications in 17 pediatric SCD patients who
underwent non-myeloablative MSD HSCT. Procedure: Medical records between
June 2013 and June 2020 were reviewed. Data was extracted from baseline
and 1,2,3, and 5-years post HSCT. Results: There were 12 females and 5
males. Follicle stimulating hormone (FSH) elevation post HSCT occurred
in 42.8%; 4 females and 2 males. All females with elevated FSH had
subsequent normalization of their values with time. FSH elevation in
males did not resolve. Post HSCT secondary amenorrhea or oligomenorrhea
was described in 4 females; however, improvement or resolution occurred
in all. One female subject with normal gonadotrophin levels post HSCT
had a successful pregnancy and live birth. Vitamin D deficiency (100%
when measured), and obese or overweight body mass index post HSCT
(41.2%) were also reported. Conclusions: A notable endocrine issue post
HSCT described in this cohort is FSH elevation. The elevation was
transient in females, and we identified one successful pregnancy,
suggesting that non-myeloablative conditioning may convey favorable
fertility outcomes compared to busulfan-based conditioning. Not all
patients had baseline endocrine evaluations or consistent post HSCT
endocrine testing. We recommend standardizing pre- and post HSCT
endocrinology assessments for this population.