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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
Author(s) -
Greystoke Brigit,
Bonanomi Sonia,
Carr Trevor F.,
Gharib Maged,
Khalid Tasneem,
Coussons Mary,
Jagani Mamta,
Naik Paru,
Rao Kanchana,
Goulden Nicholas,
Amrolia Persis,
Wynn Robert F.,
Veys Paul A.
Publication year - 2008
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2008.07064.x
Subject(s) - treosulfan , medicine , mucositis , regimen , transplantation , surgery , hematopoietic stem cell transplantation , incidence (geometry) , toxicity , conditioning regimen , physics , optics
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/m 2 /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.