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J Pediatr Hematol Oncol. 2020 Mar 20. doi: 10.1097/MPH.0000000000001782. [Epub ahead of print]

Longitudinal Description of Gonadal Function in Sickle-Cell Patients Treated With Hematopoietic Stem Cell Transplant Using Alkylator-based Conditioning Regimens.

Author information

1
Department of Pediatrics, Division of Endocrinology and Metabolism, NE Atlanta, GA.
2
Division of Pediatric Hematology/Oncology/BMT, Emory University School of Medicine, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta.
3
Department of Gynecology and Obstetrics, Division of Reproductive Endocrinology and Infertility, NE Atlanta, GA.

Abstract

OBJECTIVES:

This study describes the hormone profiles for gonadal late effects after alkylator-based hematopoietic stem cell transplant (HSCT) regimens used for sickle-cell disease (SCD).

METHODS:

This is a retrospective chart review of subjects followed in the post-HSCT clinic for sickle-cell disease. Patient demographics, pubertal development, characteristics of pre-HSCT disease severity, treatment before HSCT, conditioning regimens, presence of graft versus host disease and follicle-stimulating hormone, anti-Müllerian hormone (AMH), luteinizing hormone and testosterone were abstracted from the medical record.

RESULTS:

Forty subjects (24 female individuals) with SCD were 9 (±4.3) years old at HSCT and 7.9 years (±5.6) from HSCT. At the time of transplant, 8% of female individuals and no male individuals were pubertal and 58% of female individuals and 38% of male individuals had been treated with hydroxyurea. Post-HSCT, all of the female individuals had diminished ovarian reserve on the basis of low AMH values and 10 of the pubertal female individuals (71%) had premature ovarian insufficiency defined as follicle-stimulating hormone >40 mIU/mL ×2. There was no ovarian recovery and AMH remained very low or undetectable up to 13 years post-HSCT. In male individuals, luteinizing hormone and testosterone levels were normal for age.

CONCLUSIONS:

Post-HSCT for SCD, all female individuals had diminished ovarian reserve and most female individuals had POI, whereas male individuals had normal testosterone hormone production.

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