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Background: Beta thalassemia major and its treatment by hematopoietic stem cell transplantation can have deleterious effects on bone integrity and a main part of such effects is due to their deleterious effects on endocrine systems. So, we assessed the effects of endocrine changes during HSCT (Hematopoietic Stem Cell Transplantation) on growing bones of pediatric thalassemic patients., Methods: Bone-specific alkaline phosphatase and osteocalcin (bone formation markers), N-terminal telopeptide (NTX, bone resorption marker), calcium (Ca), phosphorus (P), alkaline phosphatase (Alk ph), parathyroid hormone (PTH), vitamin D (vit D), prolactin, luteinizing hormone (LH), follicle-stimulating hormone (FSH), thyroxine (T4), triiodothyronine (T3), thyroid-stimulating hormone (TSH), insulin-like growth factor 1 (IGF-1), testosterone (in males) or estradiol (in females), measured in 20 major thalassemic patients with mean age of 10.8±3.9 years. Parameters at the baseline (before HSCT), and 1 month and 3 months after HSCT., Results: After stem cell transplantation, changes of mean serum levels of NTX, osteocalcin, prolactin, LH, T4, IGF-1, testosterone (in males), Ca, Alk ph, PTH, and vit D were not significant, but bone specific Alk ph, P, T3, TSH, FSH and estradiol changed significantly (P=0.013, P=0.001, P=0.48, P=0.02, P=0.04 and P=0.001, respectively). After one month, there was a significant positive relationship between osteocalcine and T3 (p= 0.009). After 3 months, also, there was a significant positive relationship between osteocalcine and T3 and T4 as well as a negative one with IGF-1 (P<0.001, P<0.02 and P<0.03, respectively)., Conclusions: Endocrine disorders do not appear to have an overall positive or negative effect on bone metabolism (anabolism or catabolism) in HSCT pediatric thalassemic patients in short term (three months).

Background: Beta thalassemia major (BTM) and its treatment by hematopoietic stem cell transplantation (HSCT) may have deleterious effects on the endocrine systems. We assessed endocrine complications of HSCT in pediatric patients for 3 months., Methods: In 20 (6 female) pediatric major thalassemic patients (mean age of 10.8 ± 3.9 years old), prolactin, luteinizing hormone (LH), follicle-stimulating hormone (FSH), T4, T3, thyroid-stimulating hormone (TSH), IGF-1, testosterone (in males) or estradiol (in females) were measured as a batch at the Endocrinology and Metabolism Research Center (EMRC) of Tehran University of Medical Sciences (TUMS) laboratories before HSCT and 1 and 3 months afterwards. The cosyntropin test for all and the clonidine test for short stature patients was conducted before HSCT., Results: Before HSCT, delayed puberty and hypogonadotropic hypogonadism was found in 10% and 20% of patients, respectively. GH deficiency, low IGF1 and short stature was found in 25%, 55% and 40% of patients, respectively. Hypocortisolism, hypothyroidism and panhypopituitarism was found in 15%, 10% and 15% of patients, respectively. Prevalence of hypogonadotropic hypogonadism, low IGF1, hypothyroidism and panhypopituitarism was found in 20%, 40%, 10% and 10% of patients after 3 months, respectively (delayed puberty and short stature prevalence do not change after 3 months). HSCT caused lower T3 and estradiol and higher TSH. Corticosteroid users (15) had higher GH and lower T3 and testosterone or estradiol. Ferritin had a significant (negative) correlation with (before) prolactin and a significant correlation with T3 and T4 after HSCT. Age and acute graft-versus-host disease (GVHD) had no significant effect., Conclusion: Considering the small sample size and short duration of the study, it is difficult to reach any conclusion however it seems HSCT does not appear to have an overall positive or negative effect on prevalence of pituitary- hypothalamus axis disorders in pediatric thalassemic patients in 3 months., (© 2018 The Author(s). This is an open-access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.)

Objectives: Beta thalassemia major is a genetic hemoglobin disorder that affects bone density. The disease leads to deteriorating bone structure but can be treated with hematopoietic stem cell transplant. We aimed to assess bone mineral density changes in pediatric beta thalassemia major patients who had undergone a hematopoietic stem cell transplant compared with similarly affected patients who had not undergone a hematopoietic stem cell transplant., Materials and Methods: Forty beta thalassemia major patients, 20 transplant and 20 nontransplant, younger than 16 years of age were enrolled. The mean age of transplant patients was 8.15 years and nontransplant patients was 9.5 years (P = .242). The female:male ratio was 1:1 in both groups. None of the patients reached puberty during this study. Bone mineral density was evaluated in transplant patients before and 1 year after hematopoietic stem cell transplant. Bone mineral density of nontransplant patients also was evaluated 1 year after their initial bone mineral density test. A Norland XR-46 densitometer was used to make all bone mineral density measurements. None of the patients had a z score < -2., Results: Mean bone mineral density changes in the femur and spine during this study were 0.008 ± 0.075 g/cm2 and 0.048 ± 0.045 g/cm2 in transplant patients and 0.045 ± 0.072 g/cm2 and 0.036 ± 0.058 g/cm2 in nontransplant patients. No significant differences between bone mineral density changes in transplant and nontransplant patients were detected during the study., Conclusions: No significant effects on bone mineral density were detected in hematopoietic stem cell transplant pediatric beta thalassemia major patients compared with similarly affected nontransplant patients. Studies of longer duration may be required to identify significant changes in bone mineral density in hematopoietic stem cell transplant patients.

Background: Thalassemia major and its treatment by stem cell transplantation can have deleterious effects on bone integrity. This study assesses the adverse effects of transplantation on growing bones of pediatric thalassemic patients., Methods: Bone mineral density (BMD) of 20 patients from three thalassemia classes whose mean (SD) age was 7.4 (3.8) years were tested with a Norland XR-46 device at baseline (before transplantation), 6 and 12 months after transplantation., Results: At 6 and 12 months after transplantation we observed no significant changes in mean BMD. There were no Z-scores less than -2 among patients. Class 3 thalassemia did not negatively impact BMD. Calcium (Ca), phosphorous (P) and ferritin levels were not significantly related to patients' BMD scores. Transfusion duration and chelation therapy showed positive significant relationships to BMD (g/cm(2)), but no significant relation with the BMD Z-score. The deleterious relation between corticosteroid use and changes in BMD was not significant. In contrast, patients who developed acute graft versus host disease (aGVHD) after transplantation showed significant adverse effects on BMD of their femur (P = 0.020) and spine (P = 0.027)., Conclusion: Stem cell transplantation in pediatric thalassemic patients who do not develop aGVHD does not appear to have any significant positive or negative effects on BMD.