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

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 PieczonkaOlle Ringdén, Paul G. Schlegel, Anne Sirvent, Kim Vettenranta, Mary Eapen

Research output: Contribution to journalArticlepeer-review

54 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)711-718
Number of pages8
JournalBritish journal of haematology
Issue number5
StatePublished - Jun 1 2015

Bibliographical note

Publisher Copyright:
© 2015 John Wiley & Sons Ltd.


  • Allogeneic transplantation
  • Conditioning regimen intensity
  • Langerhans cell histiocytosis
  • Survival
  • Treatment failure


Dive into the research topics of 'Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: Outcome by intensity of conditioning'. Together they form a unique fingerprint.

Cite this