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Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning |
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| 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 |
| Institution: | 1. Great Ormond Street Hospital for Children NHS Trust, London, UK;2. Watford General Hospital, Watford, UK;3. Fred Hutchinson Cancer Research Center, Seattle, WA, USA;4. CIBMTR? (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA;5. Institute of Haematology, St. Orsola University Hospital, Azienda Ospedaliero‐Universitaria di Bologna, Bologna, Italy;6. Ospedale San Gerardo, Monza, Italy;7. EBMT Paediatric Disease Working Party, Paris, France;8. British Columbia's Children's Hospital, Vancouver, BC, Canada;9. Cook Children's Medical Center, Fort Worth, TX, USA;10. The Hospital for Sick Children, Toronto, ON, Canada;11. Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA;12. H?pital Necker‐Enfants malades, Paris, France;13. Klinik/Poliklinik für Kinderheilkunde, Münster, Germany;14. Section of Hematology‐Oncology, Department of Pediatrics, Baylor College of Medicine and the Center for Cell and Gene Therapy, Houston, TX, USA;15. Institute G. Gaslini, Genova, Italy;16. St. Jude Children's Research Hospital, Memphis, TN, USA;17. St. Anna Kinderspital, Vienna, Austria;18. H?pital d'Enfants de la Timone, Marseille, France;19. Department of Pediatrics, University of Minnesota Medical Center, Minneapolis, MN, USA;20. Department of Paediatric Oncology, Haematology & HSCT, Poznań, Poland;21. Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden;22. Centre for Allogeneic Stem Cell Transplantation, Stockholm, Sweden;23. Department of Paediatric Haematology, Oncology, Paediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany;24. H?pital Arnaud de Villeneuve, CHRU Montpellier, Montpellier, France;25. University of Helsinki, Helsinki, Finland |
| 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 treatment failure survival |