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. 2015 Jun;169(5):711-8.
doi: 10.1111/bjh.13347. Epub 2015 Mar 27.

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

Paul A Veys  1 , Vasanta Nanduri  2 , K Scott Baker  3 , Wensheng He  4 , Giuseppe Bandini  5 , Andrea Biondi  6 , Arnaud Dalissier  7 , Jeffrey H Davis  8 , Gretchen M Eames  9 , R Maarten Egeler  10 , Alexandra H Filipovich  11 , Alain Fischer  12 , Herbert Jürgens  13 , Robert Krance  14 , Edoardo Lanino  15 , Wing H Leung  16 , Susanne Matthes  17 , Gérard Michel  18 , Paul J Orchard  19 , Anna Pieczonka  20 , Olle Ringdén  21   22 , Paul G Schlegel  23 , Anne Sirvent  24 , Kim Vettenranta  25 , Mary Eapen  4
Affiliations

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

Paul A Veys et al. Br J Haematol. 2015 Jun.

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.

Keywords: Langerhans cell histiocytosis; allogeneic transplantation; conditioning regimen intensity; survival; treatment failure.

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Figures

Figure 1
Figure 1
A: The cumulative incidence of transplant-related mortality by conditioning regimen intensity B: The cumulative incidence of relapse by conditioning regimen intensity C: The probability of disease-free survival by conditioning regimen intensity D: The probability of overall survival by conditioning regimen intensity
See this image and copyright information in PMC

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