Gonadal failure among female patients after hematopoietic stem cell transplantation for non-malignant diseases

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  • Sutani Akito
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Miyakawa Yuichi
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Tsuji-Hosokawa Atsumi
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Nomura Risa
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Nakagawa Ryuichi
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Nakajima Keisuke
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan Department of Pediatrics, JA Toride Medical Center, Toride-shi, Japan
  • Maru Mitsue
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan International Nursing Development, Faculty of Nursing and Rehabilitation, Konan Women’s University, Kobe-shi, Japan
  • Aoki Yuki
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Takasawa Kei
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Takagi Masatoshi
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Imai Kohsuke
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Kashimada Kenichi
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
  • Morio Tomohiro
    Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan

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<p>In addition to malignant diseases, hematopoietic stem cell transplantation (HSCT) is also a vital option as a curative therapy for non-malignant diseases, such as immunodeficiency, and other hematological disorders. Not only for malignant diseases, but for non-malignant diseases, cytotoxic therapy of conditioning regimens are associated with high risks of adverse effects; however, clinical details regarding the long term outcomes of cytotoxic therapy for non-malignant diseases are not documented yet. To clarify the endocrinological consequences of pediatric HSCT for non-malignant disease patients, we conducted a retrospective analysis. From 1983 to 2014, 75 patients that underwent HSCT for non-malignant diseases were selected for this study. Of these, 23 patients (19 men, 4 women) were continuously followed up in our institute, with regular health check-ups for late effects. Based on a multiple linear regression analysis, the glucocorticoid treatment duration for chronic graft-versus-host disease (cGVHD) and the conditioning regimen were found to be independent predictors of growth retardation. All four female patients developed hypogonadism, and required hormone replacement therapy. The conditioning regimen for the four female patients with hypogonadism was based on the use of alkylating agents, and two female patients were treated with a reduced-intensity conditioning (RIC) regimen. Our study revealed that even the RIC regimen was toxic for the gonads in female patients, and that the survivors of both non-malignant and malignant diseases should be followed up carefully after pediatric HSCT.</p>

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