ESPE Abstracts

Introduction: As successful rates of haematopoietic stem cell transplantation (HSCT) are on the rise, late endocrine and metabolic effects of these survivors have become more prevalent.

Aims: We aimed to characterise the endocrine abnormalities observed following HSCT.

Subjects and methods: A retrospective descriptive study in paediatric post-HSCT patients (< 18 years-old) followed at an endocrine department between the years 2009 and 2019 was performed, with 157 individuals (62 females and 95 males) being identified. The reasons for HSCT were: haematopoietic pathology (42 cases of lymphoblastic leukaemia, 34 myeloblastic leukaemia, 5 lymphomas, 13 congenital anaemias and 12 cytopaenias), solid tumours (40 cases, with the most prevalent conditions being: 12 Ewing sarcomas, 9 neuroblastomas and 5 medulloblastomas) and 11 rare entities. Endocrinopathies were assessed according to the underlying diagnosis and treatment received (chemo +/- radiotherapy, surgery, type of HSCT) and regression analysis was used to ascertain association.

Results: Of all patients 58.7% presented at least one endocrine abnormality during follow-up; 15.4% of whom manifested it prior to HSCT (mainly: 5.4% short stature, 2.0% hypogonadism, 1.3% cortisol deficiency). Post HSCT patients developed primary hypogonadism (P < 0.001), had higher body mass index (P < 0.01) and iatrogenic Cushing features (P < 0.05). Endocrinopathies were most frequently developed in anaemias (82% of them) and rare entities (72% of them) and were 15% more common in females than males. A later age at HSCT [median (25^th-75^th quartiles): 9.78 years (6.25-12.25) versus 6.78 years (4.06-9.75)] was associated with endocrinopathy (P < 0.005), so that 75% of individuals transplanted ≥10 years of age developed endocrinopathies as opposed to 48.4% of those <10 years-old (P < 0.001). Similarly, pubertal Tanner stage V was associated with development of endocrinopathy (P < 0.001). Direct gonadal therapy was associated with endocrinopathies (P = 0.026) and patients who had received radiotherapy had 18% higher frequency of endocrinopathies, akin to those previously on steroids (17.4% increase). Allogenic HSCT recipients had a 7% higher rate of endocrinopathy. Although recipients of thymoglobulin or cyclophosphamide had higher incidence of endocrinopathies, there was no statistical association between conditioning agents, or their combination, and endocrinopathies. Chronic graft versus host disease was associated with endocrinopathy (P = 0.022). No significance was identified between endocrine abnormalities and follow-up duration.

Conclusions: 1. Over half of children surviving a HSCT will develop endocrine abnormalities.

2. Ascribing causality to the underlying pathology and to former therapies is challenging.

3. Following established pre- and post-HSCT protocols will enable a prompt diagnosis and treatment.