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

See, Wing-Shan; Tung, Joanna Yuet‐ling; Cheuk, Dkl; Hwang, Y.; Ip, Jing-Kun; Cheung, Pik-To; Chan, Godfrey Chi‐Fung; Ha, Shau Yin

doi:10.1038/s41409-017-0052-x

Cited by 4 publications

(4 citation statements)

References 14 publications

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“…After transplant for thalassemia, ovarian failure has been reported with a frequency ranging from 50% to 100% ( Table 4 ). 18 – 25 Here, we report that gonadal dysfunction generally resulted from the busulfan-related ovarian toxicity rather than IO which would lead to hypogonadotropic hypogonadism. In several studies of β-TM patients, older age at HSCT (>7 years) has been associated with more frequent post-transplant hypogonadism.…”

Section: Discussionmentioning

confidence: 83%

“…In several studies of β-TM patients, older age at HSCT (>7 years) has been associated with more frequent post-transplant hypogonadism. 14 , 20 , 22 , 24 , 25 This observation can be explained by the fact that the older the patient at HSCT, the higher the pre-transplant exposure to IO, but also by a possible reduced gonadal toxicity to busulfan in very young children. The pool of oocytes is limited and decreases from birth, 26 and pre-pubertal gonadal quiescence is gonadal-protective in children receiving chemotherapy.…”

Section: Discussionmentioning

confidence: 99%

“…We found a 5% rate of diabetes mellitus after transplant compared to 0-9% in previous studies on transplanted β-TM patients. 19 , 21 , 25 Potential risk factors for diabetes mellitus are: IO (because patients developing diabetes tended to be older at HSCT), conditioning therapy administration, use of corticosteroids for GvHD management.…”

Section: Discussionmentioning

confidence: 99%

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Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience

Rahal¹,

Garrigue²,

Bertrand³

et al. 2018

Haematologica

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In this retrospective study, we evaluate long-term complications in nearly all β-thalassemia-major patients who successfully received allogeneic hematopoietic stem cell transplantation in France. Ninety-nine patients were analyzed with a median age of 5.9 years at transplantation. The median duration of clinical follow up was 12 years. All conditioning regimens were myeloablative, most were based on busulfan combined with cyclophosphamide, and more than 90% of patients underwent a transplant from a matched sibling donor. After transplantation, 11% of patients developed thyroid dysfunction, 5% diabetes, and 2% heart failure. Hypogonadism was present in 56% of females and 14% of males. Female patients who went on to normal puberty after transplant were significantly younger at transplantation than those who experienced delayed puberty (median age 2.5 vs. 8.7 years). Fertility was preserved in 9 of 27 females aged 20 years or older and 2 other patients became pregnant following oocyte donation. In addition to patient’s age and higher serum ferritin levels at transplantation, time elapsed since transplant was significantly associated with decreased height growth in multivariate analysis. Weight growth increased after transplantation particularly in females, 36% of adults being overweight at last evaluation. A comprehensive long-term monitoring, especially of endocrine late effects, is required after hematopoietic stem cell transplantation for thalassemia.

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Section: Discussionmentioning

confidence: 83%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience

Rahal¹,

Garrigue²,

Bertrand³

et al. 2018

Haematologica

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“…In addition, although HSCT for BHFS is not as imminent as hemic malignancies, it should still be done as soon as possible before development of iron overload and iron‐related tissue damage. In our locality, we tend to perform allogenic HSCT for TDT patients before 8 years of age to minimize risk of subsequent development of endocrinopathies especially compromise on growth, puberty, and fertility 19 . Concerning transplant‐related mortalities and morbidities, availability of graft manipulation such as TCRαβ/CD45RA depletion lowers risk of GVHD and allows extension of donor choice to haploidentical parents, making allogeneic HSCT feasible even for patients without MSD or MUD.…”

Section: Discussionmentioning

confidence: 99%

Outcomes of allogeneic transplantation for hemoglobin Bart’s hydrops fetalis syndrome in Hong Kong

Chan

Lee

et al. 2021

Pediatric Transplantation

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Background Hemoglobin Bart's hydrops fetalis syndrome (BHFS) was once considered a fatal condition universally. Medical advances over the past three decades have resulted in increasing numbers of BHFS survivors. This retrospective review summarized local territory‐wide experience and outcomes of BHFS patients who received allogeneic hematopoietic stem cell transplantation (HSCT) in Hong Kong. Methods All BHFS patients who underwent allogeneic HSCT in Hong Kong, either in one of the two former pediatric transplant centers (Queen Mary Hospital and Prince of Wales Hospital) on or before 2019 or in the single territory‐wide pediatric transplant center (Hong Kong Children's Hospital) since 2019, from January 1, 1996, till December 31, 2020, were included. Basic demographic data, perinatal history, transplant details, long‐term outcomes, and morbidities were reviewed. Results Total five allogeneic HSCT were performed in two males and three females at a median age of 22 months, which include one 8/8 matched‐sibling bone marrow transplant, one 5/6 matched‐sibling cord blood transplant with HLA‐DR antigenic mismatch, two 12/12 matched‐unrelated peripheral blood stem cell transplant (PBSCT), and one haploidentical PBSCT with TCRαβ/CD45RA depletion from maternal donor. Neutrophil and platelet engrafted (>20 × 109/L) at a median of 15 and 22 days, respectively. All achieved near full donor chimerism at 1 month. All patients survived and remained transfusion‐independent without significant morbidities with median follow‐up duration of 10 years. Conclusion To conclude, local data demonstrated favorable outcome of allogeneic HSCT for BHFS patients, but sample number is small. Non‐directive approach in counseling and international collaboration is recommended.

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Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation

et al. 2022

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

Cited by 4 publications

References 14 publications

Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience

Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience

Outcomes of allogeneic transplantation for hemoglobin Bart’s hydrops fetalis syndrome in Hong Kong

Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation

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