Linmiao Jiang, Martin Dreyling, Olivier Hermine, Ulrich Mansmann, Jan Walewski, Vincent Ribrag, Catherine Thieblemont, Christiane Pott, Emmanuel Bachy, Pierre Feugier, Kai Hübel, Martin Schumacher, Eva Hoster
{"title":"Conditional survival of younger patients with mantle cell lymphoma: Results from a randomized phase III trial of the European MCL Network.","authors":"Linmiao Jiang, Martin Dreyling, Olivier Hermine, Ulrich Mansmann, Jan Walewski, Vincent Ribrag, Catherine Thieblemont, Christiane Pott, Emmanuel Bachy, Pierre Feugier, Kai Hübel, Martin Schumacher, Eva Hoster","doi":"10.1111/bjh.19854","DOIUrl":"https://doi.org/10.1111/bjh.19854","url":null,"abstract":"<p><p>During a fatal disease, patients often request updated information on their prognosis. After patients have already survived a certain time, conditional survival captures their future survival probability. We investigated conditional overall and failure-free survival in 473 younger mantle cell lymphoma (MCL) patients from a randomized phase III trial comparing immunochemotherapies R-CHOP and alternating R-CHOP/R-DHAP before autologous transplantation. Using conditional Kaplan-Meier method and Cox regression, we estimated subsequent survival of patients who had survived 1-8 years, considering MIPI, Ki-67, and treatment failure status. Starting at a lower level, R-CHOP patients only showed increasing subsequent survival as they survived longer (5-year conditional survival: 72% and 81% after surviving 1 and 7 years), while R-CHOP/R-DHAP patients had stable future survival over time (77% and 78%). The prognostic value of MIPI diminished after 3 years in R-CHOP patients but remained unchanged after R-CHOP/R-DHAP. Patients with treatment failure had markedly inferior survival compared with those in ongoing remission, regardless of the time survived. The longer patients remained in remission, the longer they would stay free of treatment failures. Our results enable personalized counselling for younger MCL patients by offering dynamic prognosis and underscore the importance of highly effective first-line treatment to improve survival.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Haiou Li, Vandana Sachdev, Xin Tian, My-Le Nguyen, Matthew Hsieh, Courtney Fitzhugh, Emily Limerick, Wynona Coles, Nancy Asomaning, Anna Conrey, Colin O Wu, Swee Lay Thein
{"title":"A machine learning-based workflow for predicting transplant outcomes in patients with sickle cell disease.","authors":"Haiou Li, Vandana Sachdev, Xin Tian, My-Le Nguyen, Matthew Hsieh, Courtney Fitzhugh, Emily Limerick, Wynona Coles, Nancy Asomaning, Anna Conrey, Colin O Wu, Swee Lay Thein","doi":"10.1111/bjh.19842","DOIUrl":"https://doi.org/10.1111/bjh.19842","url":null,"abstract":"<p><p>Allogeneic haematopoietic cell transplantation (HCT) with HLA-matched sibling donor remains the most established curative therapeutic option for patients with sickle cell disease (SCD). However, it is not without risks, highlighting the need for a risk stratification system. Utilizing a machine learning (ML) approach that combines clinical and imaging variables, we identified red cell distribution width and renal organ damage as important risk factors for patients undergoing HCT. This ML-based algorithm, similar to an approach previously reported for predicting mortality in patients with SCD, should be applicable to risk factor discovery in similar studies.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Comparison of HLA-haploidentical donors with post-transplant cyclophosphamide versus HLA-matched unrelated donors in peripheral blood stem cell transplantation for acute myeloid leukaemia.","authors":"Makoto Moriguchi, Hirohisa Nakamae, Mitsutaka Nishimoto, Junichi Sugita, Masamitsu Yanada, Tomomi Toubai, Yuta Hasegawa, Masayuki Hino, Tetsuya Nishida, Naoki Kurita, Masashi Sawa, Takahiro Fukuda, Atsushi Jinguji, Shuichi Ota, Ken-Ichi Matsuoka, Tetsuya Eto, Nobuhiro Hiramoto, Toshihiko Ando, Koji Kawamura, Yoshinobu Kanda, Yoshiko Atsuta, Marie Ohbiki, Hideki Nakasone, Takaaki Konuma","doi":"10.1111/bjh.19825","DOIUrl":"https://doi.org/10.1111/bjh.19825","url":null,"abstract":"<p><p>HLA-haploidentical haematopoietic cell transplantation with post-transplant cyclophosphamide (PTCy-haplo) is emerging as an effective alternative due to donor availability and safety. We conducted a nationwide retrospective study comparing the outcomes of PTCy-haplo with both anti-thymocyte globulin (ATG)-free and ATG-administered matched unrelated donors (MUD) transplantation, using peripheral blood stem cells as the first transplantation for acute myeloid leukaemia (AML). Our study showed a lower and slower haematopoietic recovery and a higher incidence of infection-related deaths after PTCy-haplo than after MUD transplantation. In addition, we revealed an increased risk of acute and chronic graft-versus-host disease (GVHD) in ATG-free MUD transplantation in comparison to PTCy-haplo. For grades III-IV acute GVHD, the hazard ratio (HR) was 2.71 (95% CI, 1.46-5.01), and for extensive chronic GVHD, the HR was 3.11 (95% CI, 2.07-4.68). There was no significant difference regarding overall survival amongst the groups. In addition, GVHD-free relapse-free survival (GRFS) was lower in ATG-free MUD transplantation than in PTCy-haplo (HR, 1.46; 95% CI, 1.17-1.82). Notably, ATG-administered MUD transplantation showed no significant difference in GRFS from PTCy-haplo, negating the advantage of PTCy. Our results suggest that PTCy-haplo could be viable for AML patients without an HLA-matched related donor.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491477","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniel Tuyet Kristensen, Mikkel Runason Simonsen, Anne Stidsholt Roug, Claus Werenberg Marcher, Andreas Due Ørskov, Anne Louise Tøllbøll Sørensen, Tarec Christoffer El-Galaly, Joachim Baech
{"title":"Outcomes among non-Western immigrant patients and Danish-born patients with acute myeloid Leukaemia: A Danish population-based cohort study.","authors":"Daniel Tuyet Kristensen, Mikkel Runason Simonsen, Anne Stidsholt Roug, Claus Werenberg Marcher, Andreas Due Ørskov, Anne Louise Tøllbøll Sørensen, Tarec Christoffer El-Galaly, Joachim Baech","doi":"10.1111/bjh.19850","DOIUrl":"https://doi.org/10.1111/bjh.19850","url":null,"abstract":"<p><p>Non-Western immigrant patients (NWIPs) may be a vulnerable population when diagnosed and treated for acute myeloid leukaemia (AML). Here we report selected quality parameters related to diagnosis, treatment, and outcome of newly diagnosed AML among NWIPs (n = 119) and Danish-born patients (DBPs) (n = 4689). No adjusted differences were observed for time-to-diagnosis, time-to-treatment, treatment allocation, rates of complete remission, early death, allogeneic stem cell transplantation, and overall survival between NWIPs and DBPs. Among patients allocated for intensive chemotherapy, NWIPs were less likely to participate in clinical trials. The findings highlight equitable AML care but underscore the need to enhance NWIP participation in clinical trials.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Risk factors and disease trajectories of recurrent immune thrombocytopenia in children.","authors":"Yuan-Ning Yang, Li-Wen Chen, Jiann-Shiuh Chen, Yung-Chieh Lin, Yun-Hsuan Yeh, Chao-Neng Cheng","doi":"10.1111/bjh.19857","DOIUrl":"https://doi.org/10.1111/bjh.19857","url":null,"abstract":"<p><p>This retrospective study aimed to analyse the course and outcome of recurrent immune thrombocytopenia (ITP) in children and to identify factors associated with recurrence. A total of 497 newly diagnosed ITP children with platelet <30 × 10<sup>9</sup>/L between January 1988 and December 2019 were included. Recurrent ITP was defined as a new event of thrombocytopenia after at least 3 months of remission without treatment. Twenty-nine (5.8%) children experienced 48 recurrent episodes. The median time from diagnosis to recurrence was 22 months. Most recurrences occurred in children aged 1.5-10 years with a recent infection history. Compared to non-recurrent ITP, children with recurrent ITP had delayed remission with lower platelets at 1 month and 3 months postdiagnosis. Multivariate analysis identified aged 1.5-10 years (hazard ratio [HR] 3.65, 95% confidence interval [CI]: 1.35-9.82) and delayed remission at 7-12 months (HR 4.04, 95% CI: 1.37-11.95) as predictors for recurrence. Most recurrent ITP patients had minor or mild symptoms, higher platelet counts, did not require treatment, and achieved remission within 12 months. The similar remission trajectories among the first and recurrent ITP, but different from the courses in the non-recurrent ITP, suggest that recurrent ITP might have a unique biological basis.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Robert Galamaga, Stephanie Johnston, Cassaundra Acosta
{"title":"Avatrombopag for the treatment of patients with chemotherapy-induced thrombocytopenia: A case series.","authors":"Robert Galamaga, Stephanie Johnston, Cassaundra Acosta","doi":"10.1111/bjh.19797","DOIUrl":"https://doi.org/10.1111/bjh.19797","url":null,"abstract":"<p><p>Management of chemotherapy-induced thrombocytopenia (CIT) is challenging, often resulting in chemotherapy treatment delays, dose reduction, and treatment interruption. Randomized trials support the potential efficacy and safety of thrombopoietin receptor agonists in CIT management. A phase III trial of avatrombopag (AVA) demonstrated increased platelet counts (PC) in patients with solid tumours experiencing CIT. To complement those findings, this case series of six patients with solid tumours and CIT is presented. Results from these cases support the clinical benefit of AVA in improving PC and reducing the impact of CIT on tumour treatment, allowing continued therapy without dose reduction or treatment interruption.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142453982","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
He Lin, Long Chang, Min Lang, Zheng-Zheng Liu, Ming-Hui Duan, Dao-Bin Zhou, Xin-Xin Cao
{"title":"Long-term follow-up of methotrexate and cytarabine in adult patients with Langerhans cell histiocytosis.","authors":"He Lin, Long Chang, Min Lang, Zheng-Zheng Liu, Ming-Hui Duan, Dao-Bin Zhou, Xin-Xin Cao","doi":"10.1111/bjh.19830","DOIUrl":"https://doi.org/10.1111/bjh.19830","url":null,"abstract":"<p><p>The optimal treatment strategy for adult Langerhans cell histiocytosis (LCH) remains unclear. Our previous study demonstrated the remarkable efficacy of combined methotrexate and cytarabine (Ara-C) [MA] therapy in patients newly diagnosed with LCH, with a median follow-up of 2 years. The present article reports long-term follow-up data spanning a median of 78 months (6.5 years) from a single-arm, single-centre, prospective phase 2 clinical trial (NCT02389400) conducted between January 2014 and December 2020. Ninety-five adults with newly diagnosed LCH exhibiting multisystem disease or multifocal single-system involvement underwent MA therapy every 35 days for six cycles. Methotrexate (1 g/m<sup>2</sup>) was administered by 24 h infusion on day 1 and AraC (0.1 g/m<sup>2</sup>) by 24 h infusion for 5 days. The primary end-point was event-free survival (EFS). The median patient age was 32 years (range 18-65 years). The overall response rate was 89.5%. Seven patients in this cohort died, and 38 experienced disease reactivation. No degenerative central nervous system diseases were observed. The estimated 6-year overall survival (OS) and EFS rates were 93.2% and 55.2% respectively. Multivariate analysis revealed that risk organ (RO) involvement at baseline (hazard ratio [HR] 6.135 [95% confidence interval (CI) 1.185-32.259]; p = 0.031) and age >40 years at diagnosis (HR 7.299 [95% CI 1.056-21.277]; p = 0.042) were associated with inferior OS. RO (HR 2.604 [95% CI 1.418-4.762]; p = 0.002) and skin (HR 2.232 [95% CI 1.171-4.255]; p = 0.015) involvement at baseline were poor prognostic factors for EFS. Regarding adverse events, four patients developed a second primary malignancy. In conclusion, the MA regimen was a valid and safe therapeutic approach for adult patients newly diagnosed with LCH.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142453991","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
A J Gerard Jansen, Minke A E Rab, Peter A W Te Boekhorst, Frank W G Leebeek
{"title":"Successful treatment with daratumumab for splenectomy refractory immune-mediated thrombotic thrombocytopenic purpura.","authors":"A J Gerard Jansen, Minke A E Rab, Peter A W Te Boekhorst, Frank W G Leebeek","doi":"10.1111/bjh.19856","DOIUrl":"https://doi.org/10.1111/bjh.19856","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142453997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Comparative outcomes of various transplantation platforms, highlighting haploidentical transplants with post-transplantation cyclophosphamide for adult T-cell leukaemia/lymphoma.","authors":"Makoto Yoshimitsu, Takashi Tanaka, Nobuaki Nakano, Koji Kato, Hiroyuki Muranushi, Masahito Tokunaga, Ayumu Ito, Jun Ishikawa, Tetsuya Eto, Satoko Morishima, Toshiro Kawakita, Hidehiro Itonaga, Naoyuki Uchida, Masatsugu Tanaka, Keiichi Akizuki, Kenji Ishitsuka, Hiroyuki Ohigashi, Shuichi Ota, Toshihiko Ando, Yoshinobu Kanda, Takahiro Fukuda, Yoshiko Atsuta, Shigeo Fuji","doi":"10.1111/bjh.19835","DOIUrl":"https://doi.org/10.1111/bjh.19835","url":null,"abstract":"<p><p>This study retrospectively compared outcomes of various allogeneic haematopoietic cell transplantation (allo-HCT) platforms in patients with adult T-cell leukaemia/lymphoma. Platforms included human leukocyte antigen (HLA)-haploidentical-related donors using post-transplant cyclophosphamide (PTCY), HLA-matched related donors (MRD), HLA-matched unrelated donors (MUD) and cord blood transplantation (CBT). Patients who underwent their first allo-HCT between 2016 and 2021 were included. Outcomes analysed were overall survival (OS), relapse and non-relapse mortality (NRM). Seven hundred patients were included (PTCY, n = 121; MRD, n = 91; MUD, n = 160; CBT, n = 328). With a median follow-up of 794 days for survivors, 2-year OS was 48.1% (PTCY), 48.8% (MRD), 48.4% (MUD) and 34.6% (CBT); the respective 2-year cumulative incidence of relapse was 37.1%, 47.5%, 33.9% and 45.1% and that of NRM was 24.2%, 19.8%, 24.7% and 27.3%. PTCY was associated with delayed platelet engraftment relative to MRD and MUD. There was no increase in the incidence of severe acute or chronic graft-versus-host disease. In the PTCY group, poor performance status was a significant predictor of inferior OS, and infused CD34+ cell numbers of less than 5 × 10<sup>6</sup>/kg were associated with delayed neutrophil and platelet engraftment. These results suggest that allo-HCT with PTCY is a safe and effective platform for patients with adult T-cell leukaemia/lymphoma.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142453987","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}