Evaluating the Patients with Thalassemia Major for Long-Term Endocrinological Complications After Bone Marrow Transplantation

Abstract

The aim of this study was to evaluate the endocrinological complications of the patients with thalassemia major (TM) who underwent bone marrow transplantation (BMT) and followed-up more than two years in our center, prospectively. "BMT group" consisted of 41 patients with TM. The mean age was 12.4 ± 5.4 years and transplantation age was mean 7.5 ± 4.9 years. Post-BMT follow-up lasted from 24 to 122 months (mean 65.07 months). Also, 32 TM patients with similar age group and same history of transfusion and chelation therapy were recruited for the study as "control (C) group". The weight SDS score after transplantation was found better than before transplantation (p = 0.010). There was a negative correlation between height SDS and BMT age (p = 0.008). The height SDS scores were better in patients whose BMT age was under seven years old compared to those older than seven years old (p = 0.02). Z-scores of femur neck and L2-4 vertebrae DEXA were decreased (p = 0.032, p = 0.0001) and incidence of insulin resistance increased (p = 0.01) in patients with increased BMT age. The risk of gonadal insufficiency was significantly lower in the patients who underwent BMT <7 years of age (p = 0.009). There was no statistically significant relationship between BMT age and complications such as hypothyroidism, hypoparathyroidism, and adrenal insufficiency. The patients with TM should be evaluated for transplantation in early stage of the disease, especially before the age of seven years. Because the BMT cannot correct the endocrinological complications of TM completely, the patients should be followed up regularly after the transplantation.

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

“…In a previous study, gonadal function is less prevalent in the HSCT group compared to the TDT group [4]. This finding was opposite to our findings.…”

“…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][2][3][4], 15-52% have short stature [1,3] and 7-19% have hypothyroidism [1,[3][4][5]. Most previous studies describe the endocrinopathies but only two have compared with TDT [2,4].…”

“…Previous studies reported limited number of diabetes mellitus or impaired glucose tolerance in post-HSCT TDT patients [3,4]. We had two patients diagnosed with diabetes mellitus, one impaired fasting glucose and two patients impaired glucose tolerance post HSCT.…”

Indicators of male fertility potential in adult patients with beta-thalassemia major: a comparative study between patients undergone allogeneic stem cell transplantation and transfusion-dependent patients

“…In this study, we could not find any difference in hypogonadism between transplanted patients with cGVHD compared with those without cGVHD. In the study conducted by Aldemir et al, they pointed out that HSCT age was a major important factor for preventing hypogonadism and early transplantation could decrease the risk of gonadal failure [23]. In a multicenter European study, comparing the age groups they reported that suspected infertility was less frequent in males who received HSCT treatment at the age of 13 or later than in those in whom treatment started at the ages of 1-12 [44].…”

“…The fertility characteristics of thalassemic patients undergone HSCT based on their age at the time of transplantation (< 13 years or ≥ 13 years) was analysed. This age classification was performed according to data available in our literature review [22,23]. In the post-HSCT group, patients' age at the time of transplantation did not significantly impact their hormonal status and sperm parameters.…”