Endocrine and Metabolic Disturbances in Survivors of Hematopoietic Stem Cell Transplantation in Childhood and Adolescence

Shalitin, Shlomit
Pertman, Lihi
Yackobovitch-Gavan, Michal
Yaniv, Isaac
Lebenthal, Yael
Phillip, Moshe
Stein, Jerry

^aThe Jesse Z. and Lea Shafer Institute of Endocrinology and Diabetes, Petach Tikva, Israel
^bDepartment of Pediatric Hematology-Oncology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel
^cSackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel

^*S. Shalitin, MD, Institute for Endocrinology and Diabetes, National Center for Childhood Diabetes, Schneider Children's Medical Center of Israel, 14 Kaplan Street, Petach Tikva 4920235 (Israel), E-Mail [email protected]

Received: July 20, 2017; Accepted: December 4, 2017; Published online: January 19, 2018

This work was performed in partial fulfillment of the thesis requirements for Lihi Pertman MD of the Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Hormone Research in Paediatrics 89(2):p 108-121, February 2018. | DOI: 10.1159/000486034

Abstract

Background/Aims:

The objective was to evaluate endocrine complications in survivors of hematopoietic stem cell transplantation (HSCT) performed during childhood.

Methods:

Endocrine dysfunction and metabolic syndrome parameters were assessed by chart review of 178 childhood HSCT survivors (median age at evaluation, 15.5 [range: 3.8-29.8] years; median follow-up, 8.5 [range: 2-23.4] years).

Results:

The following statistically significant associations were identified (p < 0.05 for all): growth hormone deficiency (17.4%) was associated with cranial/craniospinal irradiation, total body irradiation (TBI), allogeneic HSCT, and longer follow-up. Short adult stature (23.3% of patients who had attained adult height) was associated with cranial/craniospinal irradiation and, in females, with younger age at HSCT. Primary gonadal failure was more prevalent in females (52.6 vs. 24.1%), and was associated with TBI in males and with a primary diagnosis of hematological malignancy in females. Hypothyroidism (25.2%) was associated with previous neck/mediastinal irradiation. Metabolic disturbances included obesity (3.9%), type 2 diabetes (2.2%), impaired glucose tolerance (2.8%), and dyslipidemia (18.5%). Dyslipidemia was associated with a primary diagnosis of hematological malignancy, TBI, and a positive family history of dyslipidemia. Endocrine dysfunction was less frequent in patients who had received fludarabine.

Conclusions:

Patients after HSCT require long-term surveillance for the detection of endocrine and metabolic disorders. Nonmyeloablative conditioning regimens may reduce the incidence of these complications.