Therapeutic Advances in Hematology最新文献

{"title":"An occurrence of eosinophilic folliculitis and alopecia associated with a sustained complete response to mogamulizumab in Sézary syndrome: a case report","authors":"Jean-Matthieu L’Orphelin","doi":"10.1177/20406207241235777","DOIUrl":"https://doi.org/10.1177/20406207241235777","url":null,"abstract":"Mogamulizumab is a monoclonal antibody that binds to C–C chemokine receptor 4 (CCR4), initiating antibody-dependent cellular cytotoxicity. CCR4 is highly expressed in the cutaneous T-cell lymphoma subtypes mycosis fungoides and Sézary syndrome (SS), and mogamulizumab has been shown to be effective in patients with these conditions who were refractory to at least one prior systemic treatment. One of the more common adverse events encountered with mogamulizumab is rash, which may mimic disease progression and lead to premature discontinuation. Moreover, there has been some evidence to suggest that mogamulizumab-associated rash (MAR) is associated with improved outcomes in some patients, particularly those with SS. This report presents the case of a 72-year-old woman with SS, which manifested with macular and papular lesions and abnormal blood cytometry, who was treated with mogamulizumab after failure of bexarotene and photopheresis combination therapy. She achieved a complete response (CR), but experienced lymphopenia associated with histologically proven eosinophilic folliculitis (EF) of the scalp and alopecia. The EF responded well to initial topical corticosteroids, defined by regression of erythema and pustular involvement and reduction in pruritus-like symptoms, but without hair regrowth. Mogamulizumab was withdrawn after 32 cycles, but CR was maintained. To date, EF persists in the form of diffuse erythema without pustules or pruritus. A link between cluster of differentiation 4 lymphopenia and EF has previously been established; therefore, EF should be considered in patients who develop rash and lymphopenia while receiving treatment with mogamulizumab. MAR has been associated with clinical response to mogamulizumab, and this case report adds to the evidence that EF may also be associated with sustained clinical response following treatment cessation. However, regular monitoring is required to prevent a relapse of SS. Prospective studies are needed to confirm whether such an association between EF and CR following mogamulizumab exists.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"24 1","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140056493","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Interferons in the treatment of myeloproliferative neoplasms.","authors":"Pankit Vachhani, John Mascarenhas, Prithviraj Bose, Gabriela Hobbs, Abdulraheem Yacoub, Jeanne M Palmer, Aaron T Gerds, Lucia Masarova, Andrew T Kuykendall, Raajit K Rampal, Ruben Mesa, Srdan Verstovsek","doi":"10.1177/20406207241229588","DOIUrl":"10.1177/20406207241229588","url":null,"abstract":"<p><p>Interferons are cytokines with immunomodulatory properties and disease-modifying effects that have been used to treat myeloproliferative neoplasms (MPNs) for more than 35 years. The initial use of interferons was limited due to difficulties with administration and a significant toxicity profile. Many of these shortcomings were addressed by covalently binding polyethylene glycol to the interferon structure, which increases the stability, prolongs activity, and reduces immunogenicity of the molecule. In the current therapeutic landscape, pegylated interferons are recommended for use in the treatment of polycythemia vera, essential thrombocythemia, and primary myelofibrosis. We review recent efficacy, molecular response, and safety data for the two available pegylated interferons, peginterferon alfa-2a (Pegasys) and ropeginterferon alfa-2b-njft (BESREMi). The practical management of interferon-based therapies is discussed, along with our opinions on whether to and how to switch from hydroxyurea to one of these therapies. Key topics and questions related to use of interferons, such as their safety and tolerability, the significance of variant allele frequency, advantages of early treatment, and what the future of interferon therapy may look like, will be examined. Pegylated interferons represent an important therapeutic option for patients with MPNs; however, more research is still required to further refine interferon therapy.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241229588"},"PeriodicalIF":3.4,"publicationDate":"2024-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10878223/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139913474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study.","authors":"Margareth C Ozelo, Cedric Hermans, Manuel Carcao, Benoît Guillet, Joan Gu, Randy Guerra, Leilei Tang, Kate Khair","doi":"10.1177/20406207231218624","DOIUrl":"10.1177/20406207231218624","url":null,"abstract":"<p><strong>Background: </strong>Real-world data assessing treatment outcomes in patients with hemophilia A in routine clinical practice are limited.</p><p><strong>Objective: </strong>To evaluate the effectiveness and safety of octocog alfa in patients with moderate/severe hemophilia A receiving treatment in clinical practice.</p><p><strong>Design: </strong>The international Antihemophilic Factor Hemophilia A Outcome Database study is an observational, noninterventional, prospective, multicenter study.</p><p><strong>Methods: </strong>This planned interim data read-out was conducted following 7 years of observation of patients receiving octocog alfa (cut-off, 30 June 2020). The primary endpoint was joint health status, assessed by the Gilbert Score. Secondary endpoints included annualized bleeding rates (ABRs), Hemophilia Joint Health Score (HJHS), health-related quality of life, consumption, and safety. This <i>post hoc</i> analysis stratified data by hemophilia severity at baseline [moderate, factor VIII (FVIII) 1-5%; severe, FVIII <1%].</p><p><strong>Results: </strong>Of the 711 patients in this analysis, 582 (82%) were receiving prophylaxis with octocog alfa at enrollment, and 498 (70%) had severe disease. Median Gilbert Scores were higher with on-demand therapy <i>versus</i> prophylaxis and scores were comparable in moderate and severe disease. In patients receiving prophylaxis, there was an improvement in HJHS Global Gait Score over 7 years of follow-up overall and in patients with severe disease. ABRs and annualized joint bleeding rates were low across all 7 years. An ABR of zero was reported in 34-56% of prophylaxis patients <i>versus</i> 20-40% in the on-demand group. ABRs were similar in severe and moderate disease. In total, 13/702 (1.9%) patients experienced 18 treatment-related adverse events.</p><p><strong>Conclusion: </strong>These data demonstrate the long-term effectiveness and safety of octocog alfa in patients with moderate and severe hemophilia A, especially in those receiving prophylaxis. The high number of patients receiving on-demand treatment experiencing zero bleeds could be due to selection bias within the study, with patients with less severe disease more likely to be receiving on-demand treatment.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov: NCT02078427.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207231218624"},"PeriodicalIF":3.4,"publicationDate":"2024-02-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10874143/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139900430","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"When idiopathic multicentric Castleman disease meets COVID-19: a multicenter retrospective study from China.","authors":"Yi Liu, Xuejiao Yin, Dan Xu, Yunfei Lv, Li Zhu, Min Yang, Qiumei Yao, Jie Jin, Li Huang, Haitao Meng, Liangshun You","doi":"10.1177/20406207241229584","DOIUrl":"10.1177/20406207241229584","url":null,"abstract":"<p><p>Idiopathic multicentric Castleman disease (iMCD) is a rare and cytokine storm-driven inflammatory disorder. The exact cause of iMCD is still unknown, although several hypotheses have been proposed. However, regardless of the underlying cause, the ultimate result is the activation of the inflammatory pathway, which can lead to damage in multiple organs. Currently, there have been several reports highlighting the intricate link between coronavirus disease 2019 (COVID-19) and iMCD. To better understand the impact of COVID-19-induced immune storm on iMCD, we conducted a multicenter retrospective study in three hospitals in China. A total of 28 patients with iMCD were included, among whom 25 had confirmed COVID-19 infection, and we presented 4 cases that showed different disease progression after the infection of COVID-19, including 2 who did not receive any treatment for Castleman disease before. Our findings underscore the necessity of carefully monitoring iMCD patients with COVID-19 and promptly intervening to address any changes in their condition. Besides, this study also summarized the shared cytokines between COVID-19 and iMCD. Recent studies have shown promising results in treating severe COVID-19 and iMCD using tocilizumab, an interleukin-6 receptor antagonist. Therefore, it suggests that other potential cytokine storm therapy targets that have been effective in COVID-19 may also be explored for the treatment of iMCD.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241229584"},"PeriodicalIF":3.4,"publicationDate":"2024-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10865777/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139736249","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment of multiple myeloma with selinexor: a review.","authors":"Qianlei Huang, Ranran Zhao, Lu Xu, Xinbao Hao, Shi Tao","doi":"10.1177/20406207231219442","DOIUrl":"10.1177/20406207231219442","url":null,"abstract":"<p><p>Over the last 20 years, breakthroughs in accessible therapies for the treatment of multiple myeloma (MM) have been made. Nevertheless, patients with MM resistant to immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies have a very poor outcome. Therefore, it is necessary to explore new drugs for the treatment of MM. This review summarizes the mechanism of action of selinexor, relevant primary clinical trials, and recent developments in both patients with relapsed/refractory myeloma and patients with newly diagnosed myeloma. Selinexor may be useful for the treatment of refractory MM.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207231219442"},"PeriodicalIF":3.4,"publicationDate":"2024-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10771077/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139378308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes and effect of somatic mutations after erythropoiesis stimulating agents in patients with lower-risk myelodysplastic syndromes.","authors":"Juan Carlos Caballero, Julio Dávila, María López-Pavía, Esperanza Such, Teresa Bernal, Fernando Ramos, Marisa Calabuig, Jesús María Hernández Sánchez, Helena Pomares, Mercedes Sánchez Barba, María Abáigar, Bernardo González, Brayan Merchán, Reyes Sancho-Tello, Marta Callejas, Carolina Muñoz-Novas, Carlos Cerveró, Guillermo Sanz, Jesús María Hernández Rivas, María Díez Campelo","doi":"10.1177/20406207231218157","DOIUrl":"10.1177/20406207231218157","url":null,"abstract":"<p><strong>Background: </strong>Erythropoiesis stimulating agents (ESAs) are the first-line therapy in patients with lower-risk myelodysplastic syndromes (LR-MDS). Some predictive factors for ESAs response have been identified. Type and number of somatic mutations have been associated with prognosis and response to therapies in MDS patients.</p><p><strong>Objectives: </strong>The objective was to evaluate the outcomes after ESAs in patients with LR-MDS and to address the potential predictive value of somatic mutations in ESAs-treated patients.</p><p><strong>Design: </strong>Multi-center retrospective study of a cohort of 722 patients with LR-MDS included in the SPRESAS (Spanish Registry of Erythropoietic Stimulating Agents Study) study. Retrospective analysis of 65 patients with next generation sequencing (NGS) data from diagnosis.</p><p><strong>Methods: </strong>ESAs' efficacy and safety were evaluated in patients receiving ESAs and best supportive care (BSC). To assess the potential prognostic value of somatic mutations in erythroid response (ER) rate and outcome, NGS was performed in responders and non-responders.</p><p><strong>Results: </strong>ER rate for ESAs-treated patients was 65%. Serum erythropoietin (EPO) level <200 U/l was the only variable significantly associated with a higher ER rate (odds ratio, 2.45; <i>p</i> = 0.036). Median overall survival (OS) in patients treated with ESAs was 6.7 <i>versus</i> 3.1 years in patients receiving BSC (<i>p</i> < 0.001). From 65 patients with NGS data, 57 (87.7%) have at least one mutation. We observed a trend to a higher frequency of ER among patients with a lower number of mutated genes (40.4% in <3 mutated genes <i>versus</i> 22.2% in ⩾3; <i>p</i> = 0.170). The presence of ⩾3 mutated genes was also significantly associated with worse OS (hazard ratio, 2.8; <i>p</i> = 0.015), even in responders. A higher cumulative incidence of acute myeloid leukemia progression at 5 years was also observed in patients with ⩾3 mutated genes <i>versus</i> <3 (33.3% and 10.7%, respectively; <i>p</i> < 0.001).</p><p><strong>Conclusion: </strong>This large study confirms the beneficial effect of ESAs and the adverse effect of somatic mutations in patients with LR-MDS.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207231218157"},"PeriodicalIF":3.4,"publicationDate":"2024-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10768603/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139378307","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Twenty years of evolution of CML therapy: how the treatment goal is moving from disease to patient.","authors":"Domenico Russo, Michele Malagola, Nicola Polverelli, Mirko Farina, Federica Re, Simona Bernardi","doi":"10.1177/20406207231216077","DOIUrl":"https://doi.org/10.1177/20406207231216077","url":null,"abstract":"<p><p>The introduction of imatinib in 2000 opened the era of tyrosine kinase inhibitors (TKIs) for CML therapy and has revolutionized the life expectancy of CML patients, which is now quite like the one of the healthy aged population. Over the last 20 years, both the TKI therapy itself and the objectives have undergone evolutions highlighted and discussed in this review. The main objective of the CML therapy in the first 10 years after TKI introduction was to abolish the disease progression from the chronic to the blastic phase and guarantee the long-term survival of the great majority of patients. In the second 10 years (from 2010 to the present), the main objective of CML therapy moved from survival, considered achieved as a goal, to treatment-free remission (TFR). Two phenomena emerged: no more than 50-60% of CML patients could be candidates for discontinuation and over 50% of them molecularly relapse. The increased cumulative incidence of specific TKI off-target side effects was such relevant to compel to discontinue or reduce the TKI administration in a significant proportion of patients and to avoid a specific TKI in particular settings of patients. Therefore, the treatment strategy must be adapted to each category of patients. What about the patients who do not get or fail the TFR? Should they be compelled to continue the TKIs at the maximum tolerated dose? Alternative strategies based on the principle of minimal effective dose have been tested with success and they are now re-evaluated with more attention, since they guarantee survival and probably a better quality of life, too. Moving from treating the disease to treating the patient is an important change of paradigm. We can say that we are entering a personalized CML therapy, which considers the patients' age, their comorbidities, tolerability, and specific objectives. In this scenario, the new techniques supporting the monitoring of the patients, such as the digital PCR, must be considered. In the present review, we present in deep this evolution and comment on the future perspectives of CML therapy.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"14 ","pages":"20406207231216077"},"PeriodicalIF":3.4,"publicationDate":"2023-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10748527/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139032583","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Therapy for patients with chronic phase-chronic myeloid leukemia previously treated with ⩾2 tyrosine kinase inhibitors: a systematic literature review.","authors":"Ehab Atallah, Lovneet Saini, Rodrigo Maegawa, Tanvi Rajput, Regina Corbin, Ricardo Viana","doi":"10.1177/20406207221150305","DOIUrl":"https://doi.org/10.1177/20406207221150305","url":null,"abstract":"<p><strong>Background: </strong>ATP-competitive tyrosine kinase inhibitors (TKIs) are the current standard of care for patients with chronic phase-chronic myeloid leukemia (CP-CML) in the first-line and second-line (2 L) setting. Treatment after 2 L is not clearly established.</p><p><strong>Objective: </strong>The objective of this study was to summarize the available evidence to compare the efficacy and safety of interventions in the treatment of CP-CML patients who had received ⩾2 prior TKIs.</p><p><strong>Design: </strong>A systematic literature review was performed.</p><p><strong>Data source and methods: </strong>A systematic literature review (SLR) of studies published until May 2021, reporting clinical outcomes in adult patients with CP-CML who had received ⩾ 2 prior TKIs was performed. Studies were identified through the database searches via Ovid platform (Embase, MEDLINE Epub Ahead of Print, In-Process and Other Non-Indexed Citations, and Cochrane Central Register of Controlled Trials), the World Health Organization (WHO) International Clinical Trials Registry Platform (ICTRP), bibliographic search of relevant reviews, and proceedings from the previous 3 years of the key conferences in the field of oncology.</p><p><strong>Results: </strong>Our search identified 38 relevant studies. Among the identified studies of the current third-line treatments, the major molecular response (MMR) rate for ponatinib was 19.0-66.7%, 23.3-25.5% for asciminib, 19.2% for omacetaxine, and 13.2% for bosutinib at 6 months. The complete cytogenetic response (CCyR) rate was 21.4-64.8% for ponatinib, 38.7-40.8% for asciminib, 18-24.2% for bosutinib, and 16.1% for omacetaxine at 6 months.</p><p><strong>Conclusion: </strong>The findings from current SLR demonstrated the lack of data for patients with CML treated with ⩾2 TKIs. TKIs such as asciminib, ponatinib, and bosutinib are valid options for those patients. Further research is needed to identify the best treatment option for patients with CML receiving later lines of therapy.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"14 ","pages":"20406207221150305"},"PeriodicalIF":3.4,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10725100/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138802105","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy of complement inhibitors for patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis.","authors":"Jiyeon Lee, Haeseon Lee, Siin Kim, Hae Sun Suh","doi":"10.1177/20406207231216080","DOIUrl":"https://doi.org/10.1177/20406207231216080","url":null,"abstract":"<p><strong>Background: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematological disease. The development of complement inhibitors such as eculizumab, ravulizumab, and pegcetacoplan has revolutionized the management of PNH, leading to improvements in overall survival and quality of life for patients.</p><p><strong>Objectives: </strong>This systematic review aims to provide comprehensive evidence of the efficacy of complement inhibitors in relation to treatment duration.</p><p><strong>Design: </strong>This is a systematic review and meta-analysis.</p><p><strong>Data sources and methods: </strong>A thorough literature search was conducted in MEDLINE, EMBASE, and the Cochrane Library up to 3 May 2022. We included all prospective interventional studies including single-arm trials. The primary outcomes of interest were lactate dehydrogenase (LDH) levels, hemoglobin (Hb) concentrations, transfusion avoidance, and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) scores.</p><p><strong>Results: </strong>Our study included a total of 27 studies, comprising 5 randomized controlled trials and 11 single-arm trials, with a total of 912 patients with PNH. We stratified the studies according to treatment duration, based on the most frequently reported period of 26 weeks. Our analysis showed that treatment-naïve patients who received complement inhibitors had a pooled estimate of a decrease in LDH levels from baseline by -1462.0 U/L (95% CI: -1735.6 to -1188.5) for treatment ⩽26 weeks and -1696.5 U/L (95% CI: -2122.7 to -1270.2) for treatment >26 weeks. The mean Hb levels were increased by 1.4 g/dL (95% CI: 0.5-2.3) and 1.9 g/dL (95% CI: 0.7-3.1) in each group. Treatment with any complement inhibitor prevented the need for transfusion in at least 50% of patients with PNH in all treatment periods. Clinically meaningful improvements in FACIT-F were observed both before and after 26 weeks, with a pooled estimate of 6.8 (95% CI: 6.0-7.6) and 9.5 (95% CI: 7.0-12.0), respectively.</p><p><strong>Conclusion: </strong>Our findings suggest that complement inhibitors can result in positive treatment outcomes and sustained benefits for patients with PNH.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"14 ","pages":"20406207231216080"},"PeriodicalIF":3.4,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10725119/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138802102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Low-dose decitabine for previously untreated acute myeloid leukemia ineligible for intensive chemotherapy aged 65 years or older: a prospective study based on comprehensive geriatric assessment.","authors":"Ru Feng, Shuai Zhang, Jiang-Tao Li, Ting Wang, Chun-Li Zhang, Jie-Fei Bai, Lei Yang, Li-Ru Wang, Hong-Mei Jing, Hui Liu","doi":"10.1177/20406207231208979","DOIUrl":"10.1177/20406207231208979","url":null,"abstract":"<p><strong>Background: </strong>The outcome of patients with acute myeloid leukemia (AML) aged ⩾65 years is poor. Effective treatment options are limited for patients with AML who cannot tolerate intensive chemotherapy.</p><p><strong>Objectives: </strong>We aimed to evaluate the efficacy of low-dose decitabine in previously untreated patients with AML aged ⩾65 years who were ineligible for intensive chemotherapy based on a comprehensive geriatric assessment.</p><p><strong>Design: </strong>We performed a prospective, multicenter, open-label, and non-randomized study.</p><p><strong>Methods: </strong>Patients were enrolled at four centers in Beijing between 1 January 2017 and 31 December 2020. They were treated with decitabine at a dose of 6 mg/m² for 10 days. The treatment was repeated every 28 days for one cycle for a total of six cycles. The primary endpoint of our study was overall survival (OS) at the end of the first year after enrolment. The secondary endpoints included overall response rate, leukemia-free survival, relapse rate, treatment-related mortality (TRM), quality of life, safety, and transfusion dependence. Patients were continuously monitored for toxicity.</p><p><strong>Results: </strong>Overall, 47 patients (30 males and 17 females) participated in this study. The median age of the enrolled patients was 78 (range, 65-90) years. The median follow-up time was 22.2 (range, 4.6-38.8) months. Fifteen (31.9%) patients achieved complete remission (CR), 11 (23.4%) patients achieved partial remission, 3 (6.4%) patients achieved hematological improvement only, and 18 (38.3%) patients did not achieve remission. The median time to obtain CR was 2 months. The median CR was 8.5 months. Of the patients, 36 (76.6%) patients completed six cycles of treatment with low-dose decitabine, and the 1-year OS was 36.1%. According to instrumental activities of daily living scales, age, comorbidities, and albumin (IACA) scores, the median survival was 11.2 months in the unfit group and 6 months in the frail group. The 1-year OS rates in the unfit and frail groups were 49.2% and 23.4%, respectively. Grade ⩾3 non-hematological toxicity was observed in 70.2% (33/47) of the patients. TRM occurred in three patients. No early deaths occurred after treatment.</p><p><strong>Conclusion: </strong>In newly diagnosed older patients with AML whose IACA assessment was unfit or frail for standard chemotherapy, treatment with low-dose decitabine demonstrated clinical activity and good security in our study.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"14 ","pages":"20406207231208979"},"PeriodicalIF":3.4,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10685783/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138462780","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}