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Endocrine complications in patients with transfusion-dependent thalassaemia after haemopoietic stem cell transplantation

Bone Marrow Transplantation volume 53, pages 356–360 (2018)Cite this article

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Haemopoietic stem cell transplantation (HSCT) remains the only curative treatment for transfusion-dependent thalassaemia (TDT). Despite the curative intent, long-term complications like endocrinopathies can occur and cause significant morbidity. Previous studies have shown that 14.6–68% TDT post HSCT have gonadal dysfunction [1–4], 15–52% have short stature [1, 3] and 7–19% have hypothyroidism [1, 3–5]. Most previous studies describe the endocrinopathies but only two have compared with TDT [2, 4]. Most studies assessed iron overload by serum ferritin, which is not tissue-specific. MRI T2* is a more specific non-invasive measure to evaluate tissue iron in different organs [6]. With the availability of newer oral chelation, which have a high efficacy in iron chelation, better compliant and no negative impact on physical or sexual development in paediatric patients as compared to deferoxamine. We therefore took all these into consideration and carried out a study to evaluate the prevalence, cumulative incidence (CI) and risk factors of endocrinopathies of 40 TDT who underwent HSCT in 1992–2013, with ≥ 3 years of follow-up. We also randomly selected age-matched and sex-matched non-transplanted TDT controls for comparison. Demographics are summarised in Table 1.

Table 1 Demographic and characteristics of
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Authors and Affiliations

  1. Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong Special Administrative Region, Hong Kong, China

    Wing-shan Queenie See, Joanna Yuet-ling Tung, Daniel Ka-leung Cheuk, Pik-to Cheung, Godfrey Chi-Fung Chan & Shau-yin Ha

  2. Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong Special Administrative Region, Hong Kong, China

    Yu-yan Hwang

  3. Department of Radiology, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong Special Administrative Region, Hong Kong, China

    Jing-Kun Ip

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  1. Wing-shan Queenie See
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Correspondence to Wing-shan Queenie See.

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See, Ws.Q., Tung, J.Yl., Cheuk, D.Kl. et al. Endocrine complications in patients with transfusion-dependent thalassaemia after haemopoietic stem cell transplantation. Bone Marrow Transplant 53, 356–360 (2018). https://doi.org/10.1038/s41409-017-0052-x

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