IngentaConnect Treosulfan-containing regimens achieve high rates of engraftment ...

Authors: Greystoke, Brigit¹; Bonanomi, Sonia²; Carr, Trevor F.¹; Gharib, Maged¹; Khalid, Tasneem¹; Coussons, Mary¹; Jagani, Mamta²; Naik, Paru²; Rao, Kanchana²; Goulden, Nicholas²; Amrolia, Persis²; Wynn, Robert F.¹; Veys, Paul A.²

Source: British Journal of Haematology, Volume 142, Number 2, July 2008 , pp. 257-262(6)

Publisher: Blackwell Publishing

Abstract:

Summary

Treosulfan is an immuno-suppressive and myeloablative alkylating agent that has been introduced as a conditioning agent in stem cell transplantation (SCT). Most studies have been performed in adult patients with malignancy where a low incidence of regimen-related toxicity has been reported. We report the use of treosulfan in 32 consecutive children undergoing SCT for non-malignant disease. Patients received a total treosulfan dose of 36 or 42 g/m²/patient given in three daily, divided doses. A range of other conditioning agents and serotherapy was administered to patients who underwent family donor SCT (n = 11), or unrelated donor SCT (n = 21). One patient (3%) died early. Transplant morbidity was limited and mucositis was only mild. Dermatological toxicity was frequent but mild. Twenty-eight patients (87·5%) established donor cell engraftment. In 25 patients (78%) there was adequate, stable donor engraftment. Four patients have required additional transplant procedures to maintain adequate donor-derived haemopoiesis. Twenty-seven patients (84%) survive with a median follow up of 417 d. There were four late deaths due to progression of the underlying disease, graft-versus-host disease or infection. Treosulfan-based conditioning regimens achieve excellent engraftment with reduced regimen-related toxicity in children with non-malignant disease at high risk for both regimen-related toxicity and graft failure.

Keywords: conditioning; bone marrow transplantation; paediatrics; engraftment; treosulfan

Document Type: Research article

DOI: 10.1111/j.1365-2141.2008.07064.x

Affiliations: 1: Department of Blood and Marrow Transplant, Royal Manchester Children's Hospital, Manchester 2: Department of Blood and Marrow Transplant, Great Ormond Street Hospital, London, UK

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Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities