ESPE Abstracts (2019) 92 FC3.5

Evaluation of Endocrine Late Effects in Survivors of Childhood Allogeneic Hematopoietic Stem Cell Transplantation in Australia – Database from 1985 to 2011

Samantha Lai-Ka Lee1,2, Karin Tiedemann1,2, Margaret Zacharin1,2


1Royal Children's Hospital Melbourne, Melbourne, Australia. 2Murdoch Children's Research Institute, Melbourne, Australia


Background: With improved survival of childhood allogeneic hematopoietic stem cell transplant (HSCT), there is increasing need for surveillance, including assessment of endocrine late effects in this cohort1,2.

Aim: To evaluate endocrine late effects after chemotherapy and radiation in survivors of childhood allogeneic HSCT.

Methods: Multi-site evaluation via medical record review and cross-sectional questionnaires filled by patients or parents of survivors of childhood allogeneic HSCT at the Royal Children's Hospital (RCH) Melbourne between 1985 and 2011, for past and current information from RCH and adult hospitals of current attendance.

Results: 230 survivors were identified, 54.8% responded to questionnaires. Median age at transplant for malignancy was 7.7 years, with median follow up of 11.3 years. For non-malignant conditions, median age at transplant was 3.9 years and median follow up of 13.0 years. Findings were summarized in the table.

Endocrine late effectsHSCT for malignanciesHSCT for non-malignant conditions
Irradiated^Radiation-naiveIrradiated#Radiation-naive
Gonadal failure/dysfunction80.0%
(64/80)
44.4%
(28/63)
50.0%
(3/6)
22.9%
(11/48)
Siring/Having pregnancy10.2%
(6/59*)
20.6%
(7/34*)
0%
(0/5*)
9.8%
(4/41*)
Short stature35.6%
(26/73)
31.0%
(18/58)
33.3%
(1/3)
29.1%
(16/55)
Panhypopituitarism7.6%
(6/79)
0%
(0/62)
0%
(0/6)
0%
(0/58)
Growth hormone/GHRH deficiency27.5%
(22/80)
1.6%
(1/62)
16.7%
(1/6)
10.3%
(6/58)
2^ adrenal insufficiency9.0%
(7/78)
3.3%
(2/61)
0%
(0/6)
1.7%
(1/58)
Thyroid USG abnormality45.5%
(35/77)
1.7%
(1/60)
60%
(3/5)
0%
(0/58)
Thyroid malignancy16.9%
(13/77)
0%
(0/60)
20%
(1/5)
0%
(0/57)
Thyroid dysfunction44.0%
(33/75)
12.5%
(8/64)
16.7%
(1/6)
5.2%
(3/58)
Overweight33.8%
(23/68)
25.0%
(14/56)
33.3%
(1/3)
14.8%
(12/81)
Hyperlipidemia49.4%
(38/77)
15.3%
(9/59)
16.7%
(1/6)
20.7%
(12/58)
Diabetes Mellitus/Impaired fasting glucose10.7%
(8/75)
1.7%
(1/59)
0%
(0/6)
6.9%
(4/58)
Osteoporosis/Osteopenia16.9%
(13/77)
19.0%
(11/58)
0%
(0/6)
14.5%
(8/55)
Vitamin D deficiency/insufficiency16.9%
(13/77)
17.2%
(10/58)
33.3%
(2/6)
35.7%
(20/56)
^16 of 87 survivors received cranial irradiation additional to total body irradiation (TBI)
#6 survivors received TBI only
*Only those ≥18 years included in denominator

Conclusion: We confirmed endocrine late effects after childhood HSCT in a large cohort followed for up to 29 years, were frequent, seen predominantly as gonadal dysfunction, poor bone health, overweight with metabolic syndrome. Other endocrine deficiencies and thyroid malignancy were common in those who received radiation. These findings underline an urgent need for careful surveillance and very long term follow-up of HSCT survivors.

References: 1Livinalli A.Medicine(Baltimore).2019 Mar;98(12):e14921.

2Nandagopal R.Horm Res.2008;69(2):65-74.

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