Sara Lobo

and 8 more

Background: Conservative-surgery (CS) brachytherapy (BT) techniques for local therapy in bladder-prostate rhabdomyosarcoma (BP-RMS) seeks to retain organ function. We report bladder function after high-dose-rate (HDR) BT combined with targeted CS for any vesical component of BP-RMS. Procedure: Prospective cohort of all BP-RMS patients between 2014-19 receiving HDR-BT (Iridium-192, 27.5Gy in 5 fractions) with/without percutaneous endoscopic-polypectomy (PEP) or partial cystectomy (PC). Functional assessment included frequency-volume-chart, voided volumes, post-void residual, flow studies, continence status and ultrasound scanning; abnormalities triggered video-urodynamics. Results: Thirteen patients (10 male), aged 9 months to 4 years (median 23 months), presented with localised fusion-negative embryonal BP-RMS measuring 23-140mm (median 43mm) in cranio-caudal extent. After induction chemotherapy, local treatment consisted of PC+BT in three, PEP+BT in four and BT alone in six. At a median 3½ years (range 1¾-7 years) follow up, all were alive without relapse. At a median age of 6 years (4-9 years), the median bladder capacity was 86% (47%-144%) of that expected for age, including 75% (74-114%) after PC. There was no relation to radiation dose to the bladder. Complications occurred in two: one urethral stricture and one vesical decompensation in a patient with pre-existing high-grade VUR. The remaining patients are dry by day; five with anticholinergic medication for urinary urgency. Three patients are enuretic. Conclusions: Day-time dryness at a median 3½ years after CS-HDR-BT was achieved in 92%, with 85% voiding urethrally, and 62% attaining day-and-night continence aged 4-9 years. We report reduced open surgery, with minimally-invasive percutaneous surgery with HDR-BT or brachytherapy alone being suitable for many.
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.