作者
Rafał Machowicz,Felipe Suárez,W Wiktor-Jędrzejczak,Diderik-Jan Eikema,Liesbeth C. de Wreede,Henric-Jan Blok,Cecilia Isaksson,Hermann Einsele,Xavier Poiré,Suzanne van Dorp,Emmanouil Nikolousis,Jan‐Erik Johansson,Guido Kobbe,Marco Zecca,Renate Arnold,Armin Gerbitz,Jürgen Finke,José L. Díez-Martín,Francesca Bonifazi,Grant McQuaker,Stig Lenhoff,Pierre‐Simon Rohrlich,Matthias Theobald,Per Ljungman,Matthew Collin,Michael H. Albert,Gerhard Ehninger,Kristina Carlson,Kazimierz Hałaburda,Kai Lehmberg,Stefan Schönland,Ibrahim Yakoub‐Agha,Andrew R. Gennery,Arjan C. Lankester,Nicolaus Kröger
摘要
Hemophagocytic lymphohistiocytosis (HLH; hemophagocytic syndrome) is a rare syndrome of potentially fatal, uncontrolled hyperinflammation. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is indicated in primary, recurrent or progressive HLH, but information about its outcomes in the adult population is limited. We obtained data about 87 adult (≥18 years of age) patients retrospectively reported to the EBMT. The median survival time was 13.9 months. The three and five-year overall survival (OS) was 44% (95% CI 33-54%). Among 39 patients with a follow-up longer than 15 months, only three died. Relapse rate was 21% (95% CI 13-30%), while NRM reached 36% (95% CI 25-46%). Younger patients (<30 years of age) had better prognosis, with an OS of 59% (95% CI 45-73%) at three and five years vs 23% (95% CI 8-37%) for older ones. No difference in survival between reduced and myeloablative conditioning was found. To our knowledge, this is the largest report of adult HLH patients who underwent allo-HSCT. Patients who survive the first period after this procedure can expect a long disease-free survival. Both reduced intensity and myeloablative conditioning have therapeutic potential in adult HLH.