Endocrine Abstracts

Introduction: Allogeneic bone marrow transplantation (BMT) represents the only effective approach to the cure of thalassemia major, offering high rates of success especially in a pediatric setting. Endocrine complications are expected in these patients due to both primary disease and BMT process. Iron overload, desferrioxamine treatment, cytotoxic agents used in the preparative regimen, and posttransplant immunosuppression period contribute to various endocrine disorders.

Aim: To evaluate the long-term endocrine sequelae of patients with thalassemia major (TM) who underwent bone marrow transplantation (BMT) during their childhood/adolescence.

Patients and Methods: The records of patients with beta thalassemia who received bone marrow transplant during their childhood/adolescence and were followed in an Adult Endocrinology Unit were reviewed.

Results: A total of 11 patients (5 males and 6 females) who were transplanted from an HLA matched donor were identified. Their median age at bone marrow transplantation was 13(3–17) yrs. At last assessment, their median age was 28 (19–33) years old, their median height 165 (158–170) cm, their weight 65 (50–85) kg and their BMI 24.1 (17.5–29.4) kg/cm². Median follow-up duration was 15 (6–21) years.At last assessment 5/11 (45.5%) had hypothyroidism, and were on thyroxine replacement, 8/11 (72%) had osteopenia/osteoporosis, 2/11 (18.18%) had hypogonadotrophic hypogonadism and 9/11 (81.81%) had hypergonadotropic hypogonadism. All females and 3 males were on gonadal steroids. Two women had conceived spontaneously while on hormone replacement therapy and their pregnancies were both uneventful. None of the patients had adrenal insufficiency and diabetes mellitus. Two females had developed genitalia graft versus host disease (GvHD).

Conclusions: Although allogeneic BMT is known as the only definitive treatment of thalassemia major, it cannot eliminate all potential risks of endocrine dysfunction. Our results indicate that clinical findings of organ dysfunction may present or remain in the late period. Therefore, patients should be monitored for endocrine and other late complications as chronic graft-versus-host disease (GVHD) after BMT regularly.