{"doi":"10.1111/bjh.18297","title":"Outcomes of allogeneic haematopoietic cell transplantation for chronic neutrophilic leukaemia: A combined <scp>CIBMTR</scp>/<scp>CMWP</scp> of <scp>EBMT</scp> analysis","abstract":"Chronic neutrophilic leukaemia (CNL) is a rare, frequently aggressive Philadelphia (Ph)-negative myeloproliferative neoplasm (MPN). Traditional cytoreductive therapies such as hydroxyurea (hydroxycarbamide) and interferon have produced mixed results and disease responses are generally short-lived.1, 2 In a phase II study, treatment with the JAK1/2 inhibitor ruxolitinib resulted in overall response rates of 65% (n = 21) with significant reduction in CSF3R allele burden among responders.3 Despite these encouraging early results, disease progression was the main cause of death among patients treated with ruxolitinib. In a population-based analysis from the Surveillance, Epidemiology, and End Results (SEER) programme (n = 73) and the National Cancer Database (NCDB) (n = 121), the median overall survival (OS) was 1.8 and 2.2 years respectively.4 Previous retrospective studies have shown durable remission in a subset of patients who received allogeneic haematopoietic cell transplantation (allo-HCT).1, 5-8 The allo-HCT outcomes were poor when patients were transplanted in accelerated or blast-phase CNL compared to chronic-phase.9 This was an observational, multicentre analysis using the data sets of the Center for International Blood in Marrow Transplant Research (CIBMTR®) and the European Society for Blood and Marrow Transplantation (EBMT®). The protocol was approved by the institutional review boards of the National Institutes of Health Office for Human Research Protections, and the National Marrow Donor Program (NMDP) Institutional Review Board and Chronic Malignancies Working Party of the EBMT. All patients gave informed consent to use their anonymized personal information for research purposes. A total of 29 adult patients with CNL [16 (55%) from CIBMTR and 13 (45%) from the EBMT registry] who underwent their first allo-HCT between 2000 and 2018 and were reported to the CIBMTR and EBMT registries were included in the study. Cases with blast-phase or secondary acute myeloid lymphoma (AML) before transplant were not included in this analysis. The median follow-up from allo-HCT was 71 (range: 25–161) months for the entire cohort. The median patient age was 58 (range: 33–72) years and the median time from diagnosis of CNL to allo-HCT was 11 (range: 3–65) months. The disease status at allo-HCT was reported to be no response/stable disease in 11 (38%) patients and complete remission (CR) in four (14%) patients. This information was missing in four (14%) patients. Among the 25 patients with available information, splenomegaly was reported in eight (28%) patients and the use of hydroxyurea was reported in 19 (66%) patients. Other reported pretransplant therapies included hypomethylating agent (n = 4), tyrosine kinase inhibitor (n = 7) and ruxolitinib (n = 7). Data on somatic mutations was only available in a subset (n = 9) of patients. The following somatic mutations were reported in the database: CSF3R (n = 9), ASXL1 (n = 4), DNMT3A (n = 1), IDH2 (n = 1), SETBP1 (n = 4), SRSF2 (n = 2), CUX1 (n = 1), SF3B1 (n = 3) RUNX1 (n = 2). JAK2 was tested and negative in 17 (59%) patients. Please refer to Table S2 (Supporting information) for full details on genetic abnormalities. Most transplants were performed using a human leukocyte antigen (HLA)-identical sibling (n = 12, 41%) or 8/8 unrelated donor (n = 10, 34%) with a peripheral blood stem cell (PBSC) graft (n = 27, 93%). The myeloablative conditioning (MAC) regimen was used in 14 (48%) patients and calcineurin-inhibitor-based graft-versus-host disease (GVHD) prophylaxis was used in 22 (76%) patients. Table 1 shows the detailed baseline characteristics. The outcomes were censored at 48 months from allo-HCT, based on median follow-up duration of the CIBMTR cohort. The non-relapse mortality (NRM) was 10.3% [95% confidence interval (CI): 2–24.1] at one year and 13.8% (95% CI: 3.7–28.9) at four years after allo-HCT. Most relapses were seen within one year of allo-HCT with one- and four-year rela","journal":"British Journal of Haematology","year":2022,"id":290075,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":4,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9481,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2022-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":972096,"name":"Aleksandar Radujkovic","orcid":"0000-0002-8543-5016","position":1,"is_corresponding":false},{"id":408356,"name":"Noel Estrada‐Merly","orcid":null,"position":2,"is_corresponding":false},{"id":972526,"name":"Tiarlan Sirait","orcid":null,"position":3,"is_corresponding":false},{"id":348722,"name":"Soyoung Kim","orcid":"0000-0003-1404-0575","position":4,"is_corresponding":false},{"id":972097,"name":"Juan Carlos Hernández‐Boluda","orcid":"0000-0002-4289-3113","position":5,"is_corresponding":false},{"id":972098,"name":"Tomasz Czerw","orcid":"0000-0003-3108-4035","position":6,"is_corresponding":false},{"id":972099,"name":"Patrick Hayden","orcid":"0000-0003-1374-4503","position":7,"is_corresponding":false},{"id":345197,"name":"Ankit Kansagra","orcid":"0000-0001-6901-0782","position":8,"is_corresponding":false},{"id":282399,"name":"Vincent T. Ho","orcid":"0000-0001-6839-3996","position":9,"is_corresponding":false},{"id":196670,"name":"T Nishihori","orcid":"0000-0002-2621-7924","position":10,"is_corresponding":false},{"id":972100,"name":"Paul Shaughnessy","orcid":"0000-0003-4023-3864","position":11,"is_corresponding":false},{"id":284155,"name":"Bart L. Scott","orcid":"0000-0001-9620-7839","position":12,"is_corresponding":false},{"id":972527,"name":"R. Nakamura","orcid":null,"position":13,"is_corresponding":false},{"id":348679,"name":"Betül Oran","orcid":"0000-0003-1264-5968","position":14,"is_corresponding":false},{"id":284139,"name":"Mohamed A. Kharfan‐Dabaja","orcid":"0000-0001-7394-5185","position":15,"is_corresponding":false},{"id":284148,"name":"Bipin N. Savani","orcid":"0000-0002-3304-9965","position":16,"is_corresponding":false},{"id":972528,"name":"Donal P. McLornan","orcid":null,"position":17,"is_corresponding":false},{"id":972101,"name":"Ibrahim Yakoub‐Agha","orcid":"0000-0003-4524-8782","position":18,"is_corresponding":false},{"id":122972,"name":"Wael Saber","orcid":"0000-0002-6544-5815","position":19,"is_corresponding":false},{"id":521113,"name":"Bhagirathbhai Dholaria","orcid":"0000-0003-2371-3655","position":0,"is_corresponding":true}],"reference_count":10,"raw_metadata":null,"created_at":"2026-07-19T00:30:26.667578Z","pmid":"35658101","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}