Patient-reported late effects of single fraction total body irradiation
for non-malignant haematological disease transplant conditioning
Abstract
Introduction: Patients with severe complications of non-malignant
haematological disease are considered as candidates for curative
treatment with an allogenic bone marrow transplant (ABMT). A
non-myeloablative conditioning regimen is used; consisting of an
alkylating agent and single fraction total body irradiation (SFTBI) at a
dose of 2-4.5 Gy (dose rate 150mu/min). This is distinct from high dose
fractionated total body irradiation (TBI) used in a myeloablative
conditioning regimen; for which the late effects are well documented.
There is however no dedicated study on the late effects associated with
low dose SFTBI. Methods: We undertook a single institution study
focusing on patient reported outcomes after SFTBI (January 2003 –
January 2019) delivered more than 1-year previously, prior to an AMBT in
patients aged under 16-years for non-malignant haematological
conditions. A 19-point questionnaire was conducted with study subjects
over the phone. The primary outcome was late effects as reported by
patients. Secondary outcomes were patient demographics. Results: Fifty
patients were screened, 31 were invited to take part and 24 consented to
participate. Pulmonary toxicity was the most common visceral effect
reported (5 patients), followed by kidney (3) and cardiac (2). No
patients reported cataracts, diabetes or secondary malignancy. Two
patients were on sex hormone replacement although no evidence of female
menstrual delay was demonstrated. The majority (21) were enrolled in
mainstream schools. Conclusion: Late effects do occur after SFTBI, but
are mild and occur less frequently compared to high dose TBI. The
consent process with children/parents prior to SFTBI should reflect
this.