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Gonadal function of beta-thalassemics following stem cell transplantation conditioned with myeloablative and reduced intensity regimens

  • Preamrudee Poomthavorn EMAIL logo , Pongpan Chawalitdamrong , Suradej Hongeng , Pat Mahachoklertwattana , Samart Pakakasama , Patcharin Khlairit and La-or Chailurkit
Published/Copyright: May 24, 2013
Journal of Pediatric Endocrinology and Metabolism
From the journal Journal of Pediatric Endocrinology and Metabolism Volume 26 Issue 9-10

Abstract

Gonadal dysfunction is a complication following stem cell transplantation (SCT). There have been no reports of gonadal function in stem-cell-transplanted thalassemic survivors who received a reduced intensity conditioning regimen (RIC). We evaluated gonadal function in 47 β-thalassemic patients following SCT with either myeloablative or reduced intensity regimen. Thirty-six patients received a myeloablative regimen, the remaining 11 patients had an RIC regimen. Their median (range) age was 13.2 (5.9–25.8) years. There were 29 patients (62%) with gonadal dysfunction (26 with primary gonadal dysfunction and three with gonadotropin deficiency). Comparisons between patients who received myeloablative and RIC regimens, revealed no differences in gonadal dysfunction (56% vs. 82%, p=0.113, respectively). In conclusion, our study demonstrated high frequency of gonadal dysfunction in these patients. Even after receiving RIC, gonadal dysfunction was very common. To our knowledge, this study is the first to report gonadal function in children and adolescents with β-thalassemia disease who were pre-transplanted with RIC.

Keywords: gonadal failure; gonadal function; thalassemia disease; transplantation
Corresponding author: Preamrudee Poomthavorn, MD, Faculty of Medicine Ramathibodi Hospital, Department of Pediatrics, Mahidol University, Bangkok 10400 Thailand, Phone: +662 201-1394, Fax: +662 201-1850, E-mail:

This study was supported by a Research Grant from the Faculty of Medicine Ramathibodi Hospital, Mahidol University.

Conflict of interest statement

Financial disclosure: None.

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Received: 2012-12-12
Accepted: 2013-4-18
Published Online: 2013-05-24
Published in Print: 2013-10-01

©2013 by Walter de Gruyter Berlin Boston

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https://doi.org/10.1515/jpem-2012-0399
Keywords for this article
gonadal failure; gonadal function; thalassemia disease; transplantation

Articles in the same Issue

  1. Masthead
  2. Masthead
  3. Review article
  4. Tamoxifen therapy for the management of pubertal gynecomastia: a systematic review
  5. Images in pediatric endocrinology
  6. Solitary median maxillary central incisor, a clinical predictor of hypoplastic anterior pituitary, ectopic neurohypophysis and growth hormone deficiency
  7. A rare case of granuloma annulare in a 6-year-old child with type 1 diabetes
  8. Original articles
  9. The arginine stimulation test: timing of peak is not a helpful parameter in the diagnosis of growth hormone deficiency
  10. The relationship between adiposity and stature in prepubertal children with celiac disease
  11. Influence of micronutrient status and socioeconomic gradient on growth indices of 2–18-year-old Indian girls
  12. Phosphotyrosine phosphatases in GH-stimulated skin fibroblasts from children with idiopathic short stature
  13. The relation between salivary cortisol and the metabolic syndrome score in girls
  14. Association of vitamin D concentrations with adiposity indices among preadolescent children in Korea
  15. Association of serum resistin levels with metabolic syndrome and early atherosclerosis in obese Chinese children
  16. Characteristics of infants at risk of hypoglycaemia secondary to being ‘infant of a diabetic mother’
  17. Parental dyadic coping in families of children and adolescents with type 1 diabetes
  18. Pigmentary hypertrichosis and non-autoimmune insulin-dependent diabetes mellitus (PHID) syndrome is associated with severe chronic inflammation and cardiomyopathy, and represents a new monogenic autoinflammatory syndrome
  19. Effect of insulin therapy on IGF-1 level in children with new-onset type 1 diabetes mellitus: a comparison between DKA and non-DKA
  20. Mutation analysis of the SRD5A2, AR and SF-1 genes in 52 Chinese boys with hypospadias
  21. Sexual development in prepubertal obese boys: a 4-year longitudinal study
  22. Genetic diagnosis of one family with incomplete clinical data
  23. Patient reports
  24. Complete androgen insensitivity syndrome and discordant Müllerian remnants: two cases with novel mutation in the androgen receptor
  25. Psychosexual outcomes in three siblings with partial androgen insensitivity syndrome: impact of nature versus nurture
  26. Ambiguous genitalia in a 48, XXYY newborn: a casual relationship or a coincidence?
  27. Gonadal function of beta-thalassemics following stem cell transplantation conditioned with myeloablative and reduced intensity regimens
  28. Significant testicular enlargement in a 6.5-year-old boy with monorchism and testicular microlithiasis
  29. Report of the first case of precocious puberty in Rett syndrome
  30. Type II vitamin D-dependent rickets with diabetic ketoacidosis
  31. Pituitary gigantism presenting with depressive mood disorder and diabetic ketoacidosis in an Asian adolescent
  32. A rare association between Rathke’s cyst and hypophysitis in a patient with delayed sex development and growth failure
  33. Diabetes mellitus with Laron syndrome: case report
  34. Wegener granulomatosis as an uncommon cause of panhypopituitarism in childhood
  35. Rare cases of autoimmune hypothyroidism in young children
  36. Cushing syndrome related to leukemic infiltration of the central nervous system: a case report and a possible role of LIF
  37. Early presentation of bilateral gonadoblastomas in a Denys-Drash syndrome patient: a cautionary tale for prophylactic gonadectomy
  38. Identification of a novel homozygous mutation (S144I) in a Malay patient with maple syrup urine disease
  39. Virilization in two pre-pubertal children exposed to topical androgen
  40. A family with Camurati-Engelman disease. The role of the missense p.R218C mutation in TGFB1 in bones and endocrine glands
  41. Short communications
  42. Plasma ceramides are elevated in female children and adolescents with type 2 diabetes
  43. Assessment of contractility and myocardial function in small and appropriate for gestational age premature neonates using the stress-velocity relationship and tissue Doppler imaging immediately after birth
  44. Letter to the Editors
  45. Homeostasis model assessment of insulin resistance may not be reliable to evaluate insulin sensitivity in young obese children
  46. Type 1 diabetes in women with endometriosis: what is the risk of occurrence?
  47. How should we manage vitamin D-deficient adolescents?
  48. Should neonatal hyperparathyroidism associated with mucolipidosis II/III be treated pharmacologically?a
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