Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning.

Abstract

Patients with Langerhans cell histiocytosis (LCH) refractory to conventional chemotherapy have a poor outcome. There are currently two promising treatment strategies for high-risk patients: the first involves the combination of 2-chlorodeoxyadenosine and cytarabine; the other approach is allogeneic haematopoietic stem cell transplantation (HSCT). Here we evaluated 87 patients with high-risk LCH who were transplanted between 1990 and 2013. Prior to the year 2000, most patients underwent HSCT following myeloablative conditioning (MAC): only 5 of 20 patients (25%) survived with a high rate (55%) of transplant-related mortality (TRM). After the year 2000 an increasing number of patients underwent HSCT with reduced intensity conditioning (RIC): 49/67 (73%) patients survived, however, the improved survival was not overtly achieved by the introduction of RIC regimens with similar 3-year probability of survival after MAC (77%) and RIC transplantation (71%). There was no significant difference in TRM by conditioning regimen intensity but relapse rates were higher after RIC compared to MAC regimens (28% vs. 8%, P = 0·02), although most patients relapsing after RIC transplantation could be salvaged with further chemotherapy. HSCT may be a curative approach in 3 out of 4 patients with high risk LCH refractory to chemotherapy: the optimal choice of HSCT conditioning remains uncertain.

• Authors: Paul A Veys, Vasanta Nanduri, K Scott Baker, Wensheng He, Giuseppe Bandini, Andrea Biondi, Arnaud Dalissier, Jeffrey H Davis, Gretchen M Eames, R Maarten Egeler, Alexandra H Filipovich, Alain Fischer, Herbert Jürgens, Robert Krance, Edoardo Lanino, Wing H Leung, Susanne Matthes, Gérard Michel, Paul J Orchard, Anna Pieczonka, Olle Ringdén, Paul G Schlegel, Anne Sirvent, Kim Vettenranta, Mary Eapen
• Journal: British journal of haematology (Br J Haematol) Vol. 169 Issue 5 Pg. 711-8 (Jun 2015) ISSN: 1365-2141 [Electronic] England
• PMID: 25817915 (Publication Type: Journal Article, Research Support, N.I.H., Extramural)
• Copyright: © 2015 John Wiley & Sons Ltd.

Topics

• Adolescent
• Adult
• Child
• Child, Preschool
• Female
• Graft vs Host Disease (etiology, prevention & control)
• Hematopoietic Stem Cell Transplantation (adverse effects)
• Histiocytosis, Langerhans-Cell (mortality, pathology, therapy)
• Humans
• Infant
• Male
• Retrospective Studies
• Transplantation Conditioning
• Transplantation, Homologous
• Treatment Outcome
• Young Adult