Late effects after hematopoietic stem cell transplantation in patients with HLH: A Histiocyte Society, PDWP, IEWP, and TCWP EBMT Study
Year: 2025
Type: article
Study design: Observational Study
Abstract: BACKGROUND: Hematopoietic stem cell transplantation (HCT) is the only curative treatment in primary hemophagocytic lymphohistiocytosis (pHLH). However, HCT is associated with a wide range of late effects (LEs). OBJECTIVE: We sought to characterize the long-term outcome and LEs following HCT in pHLH. METHODS: A total of 274 children with pHLH from the European Society for Blood and Marrow Transplantation registry who underwent allogeneic HCT between 2004 and 2015 were included. Multivariable logistic regression models were used to evaluate the adjusted impact of baseline variables on central nervous system and hormonal LEs, respectively. RESULTS: A broad spectrum of LEs was identified, with neurologic (31%) and hormonal (34.8%) complications being the most prevalent. Chemotherapy (HLH-1994/HLH-2004) before HCT was identified as a significant risk factor for endocrinological LEs (P = .03), highlighting a novel aspect not previously reported. The presence of neurologic abnormality at diagnosis was an independent risk factor for neurologic LEs (P < .001) as was incomplete remission status at the time of HCT (P = .04). CONCLUSIONS: HCT has significantly improved survival in patients with pHLH. However, survivors still face significant risks of LEs.
Authors: Kim Ramme, AnnaCarin Horne, Karin Beutel, Jacques-Emmanuel Galimard, Ali Abdallah Alahmari, Giorgio Ottaviano, Despina Moshous, Savas Kansoy, Zohreh Nademi, Maura Faraci, Mikael Sundin, Franca Fagioli, Michael H. Albert, Petr Sedlacek, Yves Bertrand, Franco Locatelli, Catherine Paillard, Karin Mellgren, Ingo Müller, Johann Greil, Adriana Balduzzi, Mary Slatter, Arnaud Dalissier, Krzysztof Kalwak, Arjan Lankester, Benedicte Neven, K. Scott Baker, Kanchan Rao, Selim Corbacioglu
Institutions: Uppsala University Hospital, Karolinska University Hospital, Karolinska Institutet, München Klinik Schwabing, München Klinik, Technical University of Munich, Inserm, Sorbonne Université, Assistance Publique – Hôpitaux de Paris, Hôpital Saint-Antoine, Centre de Recherche Saint-Antoine, King Faisal Specialist Hospital & Research Centre, University College London, Hôpital Necker-Enfants Malades, Laboratoire d'hématologie, Ege University, Great North Children's Hospital, Istituto Giannina Gaslini, Ospedale Regina Margherita, University of Turin, LMU Klinikum, Ludwig-Maximilians-Universität München, University Hospital in Motol, Institut d’Hématologie et d’Oncologie Pédiatrique, Bambino Gesù Children's Hospital, Hôpitaux Universitaires de Strasbourg, Université de Strasbourg, Sahlgrenska University Hospital, University Medical Center Hamburg-Eppendorf, Heidelberg University, University of Milano-Bicocca, Royal Victoria Infirmary, University of Wrocław, Wroclaw Medical University, Leiden University Medical Center, Willem-Alexander Kinderziekenhuis, Fred Hutch Cancer Center, National Health Service, Great Ormond Street Hospital, University of Regensburg
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,900
Funder: Histiocytosis Association
OpenAlex ID: W4414625345