Leukemia Research Reports最新文献

{"title":"LUSPATERCEPT IN TRANSFUSION-DEPENDENT MYELODYSPLASTIC SYNDROMES: REAL WORLD DATA","authors":"Y. Gong ,&nbsp;L. Zhou ,&nbsp;T. Zhang ,&nbsp;D. Xu ,&nbsp;Y. Zhang ,&nbsp;J. Shi ,&nbsp;J. Li ,&nbsp;G. He","doi":"10.1016/j.lrr.2024.100443","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100443","url":null,"abstract":"<div><h3>Introduction</h3><p>Luspatercept reduced the severity of anemia in transfusion-dependent lower risk MDS with ring sideroblasts (RS) in the MEDALIST trial. Here we present real-world data in a prospective multicenter registry study of MDS patients treated with luspatercept (ChiCTR2300071857). Patients who were not enrolled in the MEDALIST trial were also evaluated, including those of higher IPSS-R risk, without ring sideroblasts, or failed to prior hypomethylating agent (HMA) therapy.</p></div><div><h3>Methods</h3><p>Eligible patients were ≥ 18 years old and were RBC transfusion-dependent (defined as ≥2 units of RBC transfusion per 8 weeks). Primary endpoint was RBC transfusion independence (RBC-TI) for ≥8 weeks. Secondary endpoints are hematological improvement-erythroid (HI-E, defined as &gt;50% reduction in RBC transfusion), neutrophil (HI-N) and platelet (HI-P) per the IWG 2006 criteria.</p></div><div><h3>Results</h3><p>From June 2022 to August 2023, 25 patients were treated with luspatercept for a median of 7 cycles <strong>(Table 1)</strong>. The median follow-up time was 19.4 weeks. The rate of RBC-TI was 36%. The median duration of RBC-TI was 227 [IQR: 129-324] days. The rates of HI-E, HI-N, HI-P were 52% (13/25), 21% (3/14) and 33% (5/15), respectively. Multi-variant analysis revealed that lower transfusion burden (&lt;6 u/8w) favored higher RBC-TI rate (OR: 0.041, 95% CI: 0.003∼0.666, p=0.025), while other characteristics such as IPSS-R risk, presence of RS or <em>SF3B1</em> and prior therapy had no impact on the outcome <strong>(Table 1)</strong>.</p></div><div><h3>Conclusions</h3><p>Our real-world data confirms the clinical benefit of luspatercept observed in the MEDALIST trial. Luspatercept retains efficacy in patients without RS, or failed to prior HMA therapy.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100443"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000335/pdfft?md5=d52127e1c5b3f5d6660065b8b54c179f&pid=1-s2.0-S2213048924000335-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103892","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Myeloid neoplasms in inflammatory bowel disease: A case series and review of the literature","authors":"David M. Mueller ,&nbsp;Daniel I. Nathan ,&nbsp;Angela Liu ,&nbsp;John Mascarenhas ,&nbsp;Bridget K. Marcellino","doi":"10.1016/j.lrr.2024.100458","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100458","url":null,"abstract":"<div><p>Patients with inflammatory bowel disease (IBD) are exposed to chronic systemic inflammation and are at risk for secondary malignancies. Here we review the literature on the risk of myeloid neoplasms (MN) in IBD and present the disease profiles of patients at a single institution with IBD who later developed MN, comparing them to those in the literature. No IBD characteristic was found to associate with MN disease severity, including the previously-identified association between MNs and thiopurine exposure. Of the somatic mutations identified in out cohort's MN, mutations in <em>TET2</em> were most prevalent, followed by <em>FLT3-ITD, BCR-ABL</em>, and <em>NPM1</em> mutations.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100458"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000487/pdfft?md5=772ffe0cac241176bb4b87667c2b6fd1&pid=1-s2.0-S2213048924000487-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140347118","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A rare case of Blastic plasmacytoid dendritic cell neoplasm with gynecologic presentation","authors":"Marzouk Khouloud ,&nbsp;Slama Nader ,&nbsp;Bellalah Ahlem ,&nbsp;Safra Ines ,&nbsp;Hafsi Montacer ,&nbsp;Chabbeh Rayhan","doi":"10.1016/j.lrr.2024.100462","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100462","url":null,"abstract":"<div><p>Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy characterized by the proliferation of plasmacytoid dendritic cells with a blast-like appearance. It usually presents in elderly people, and clinical manifestations include nodular blue-violet skin lesions, bone marrow infiltration and, less frequently, extramedullary involvement. Gynecological manifestation (breast mass and exocervical lesion) is an unusual and rare presentation.</p><p>Herein, we report the case of a 51-year-old woman patient who presented with a history of a rapidly growing and bleeding breast mass, along with a decline in general health. Notably, the disease had multifocal involvement, affecting the breast, uterine cervix, and cervical lymphadenopathy.</p><p>Biopsies were performed on the breast mass and cervical lesion. Histopathological examination showed a diffuse lymphoid proliferation. The neoplastic cells show immunoreactivity for CD45 and CD56.</p><p>The myelogram showed a 50 % excess of blasts with a heterogeneous appearance with the presence of cells that could suggest dendritic plasmacytoid cells. Bone marrow immunophenotyping showed the presence of blast-like cells that were positive for CD4, CD56, CD123, which supported the diagnosis of BPDCN.</p><p>Despite initiating chemotherapy, the patient's condition rapidly deteriorated, highlighting the aggressive nature of BDCP. This case underscores the importance of early detection and the need for further research to improve outcomes for this rare condition.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100462"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000529/pdfft?md5=74f9636dea36ae93c2e73ad29a55bdc8&pid=1-s2.0-S2213048924000529-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141090650","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Indolent infections in patients with hematologic malignancy: A single-center experience screening for tuberculosis and strongyloidiasis prior to cytotoxic therapy in Boston","authors":"K.A. Reifler ,&nbsp;T. Francoeur Smith ,&nbsp;G. Bodanapu ,&nbsp;M. Fagan ,&nbsp;D.L. Bourque ,&nbsp;J.M. Sloan","doi":"10.1016/j.lrr.2024.100489","DOIUrl":"10.1016/j.lrr.2024.100489","url":null,"abstract":"<div><div>Individuals with hematologic malignancy have increased risk of latent tuberculosis infection (LTBI) reactivation and <em>Strongyloides stercoralis</em> (SS) dissemination. However, screening prior to chemotherapy or corticosteroids is not routine. We conducted a LTBI and SS screening intervention amongst patients with hematologic malignancies. Over one-third immigrated from countries with high TB incidence and SS prevalence, and some had additional risk factors (HIV, HTLV-1, eosinophilia). The intervention increased screening rates but universal screening was not achieved. Screening positivity was similar pre- and post-intervention. We demonstrate a population with increased indolent infection reactivation risk. Specific infectious screening guidelines are needed to prevent significant morbidity.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100489"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142744789","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Management of patients with concurrent clonal plasma cell and myeloid disorders: A single center descriptive case series","authors":"Michael J. Hochman ,&nbsp;Gloria F. Gerber ,&nbsp;Philip H. Imus ,&nbsp;Syed Abbas Ali ,&nbsp;Amy E. DeZern","doi":"10.1016/j.lrr.2024.100469","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100469","url":null,"abstract":"<div><p>Both clonal plasma cell and myeloid disorders occur more frequently with age. Patients with concurrent clonal plasma cell and myeloid disorders (CPCMD) can present clinical and therapeutic challenges. In this single-institution cohort of patients with CPCMD (<em>n</em> = 18), we abstracted clinically relevant themes. A majority of patients (12/18) were treated with clone-directed therapies and three received treatment targeting both clones. Treatment of clones with targetable genetic lesions or those causing end-organ complications should be prioritized. Simultaneous treatment of both clones can be safe but is best done in a stepwise manner. Further study of patients with dual clonal processes is warranted.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100469"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000591/pdfft?md5=4065ce5b8b828eb534a8db9898f07b60&pid=1-s2.0-S2213048924000591-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141605705","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A comprehensive case study on successful multimodal therapy in philadelphia chromosome-positive acute myeloid leukemia with NPM1 and IDH2 mutations","authors":"Syed Muhammad Waqar Haider ,&nbsp;Mehwish Zehra ,&nbsp;Nikesh N Shah ,&nbsp;Eduardo M Sotomayor ,&nbsp;David M Swoboda","doi":"10.1016/j.lrr.2024.100461","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100461","url":null,"abstract":"<div><p>A 67-year-old female came to Tampa General Hospital with Philadelphia chromosome-positive (Ph+) acute myeloid leukemia (AML) featuring an intriguing combination of mutations, including <em>NPM1</em> and <em>IDH2</em> mutations. Novel combination therapy with azacitidine, venetoclax and ponatinib allowed her to successfully achieve a complete response (CR) and undergo an allogeneic hematopoietic stem cell transplant (HSCT). This case report provides an overview of her clinical course, emphasizing the significance of integrated therapy and the challenges associated with balancing treatment for AML. It also underscores the importance of a multidisciplinary approach and careful monitoring of patients with complex hematologic conditions.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100461"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000517/pdfft?md5=2596c4784f9889a364c2caaf23574cb6&pid=1-s2.0-S2213048924000517-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140822895","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CLINICAL IMPLICATIONS OF COPY NUMBER ALTERATION DETECTION USING PANEL-BASED NEXT-GENERATION SEQUENCING DATA IN MYELODYSPLASTIC SYNDROME","authors":"S.-H. Jung ,&nbsp;Y.-J. Chung","doi":"10.1016/j.lrr.2024.100426","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100426","url":null,"abstract":"<div><h3>Introduction</h3><p>Recurrent somatic mutations are found in about 90 % of the MDS patients, and some of the mutations are known to have a prognostic value at various clinical stages of myelodysplastic syndrome (MDS). However, there are no specific guidelines for the assessment of CNAs, and the CNA test is not included in standard practice in the diagnosis and prognostication of MDS. Thus, there is a need to evaluate whether the detection of CNAs, in addition to genetic mutations, has clinical significance. In this study.</p></div><div><h3>Methods</h3><p>Targeted deep sequencing of 28 well-known MDS-related genes was performed for 266 patients with MDS. DNA copy number was estimated using the targeted deep sequencing data.</p></div><div><h3>Results</h3><p>Overall, 215 (80.8 %) patients were found to have at least one somatic mutation; 67 (25.2 %) had at least one CNA; 227 (85.3 %) had either a somatic mutation or CNA; and 12 had CNA without somatic mutations. Considering the clinical variables and somatic mutations alone, multivariate analysis demonstrated that sex, revised International Prognostic Scoring System (IPSS-R), and NRAS and TP53 mutations were independent prognostic factors for overall survival. For AML-free survival, these factors were sex, IPSS-R, and mutations in NRAS, DNMT3A, and complex karyotype/TP53 mutations. When we consider clinical variables along with somatic mutations and CNAs, genetic alterations in TET2, LAMB4, U2AF1, and CBL showed additional significant impact on the survivals.</p></div><div><h3>Conclusions</h3><p>In conclusion, our study suggests that the concurrent detection of somatic mutations and targeted CNAs may provide clinically useful information for the prognosis of MDS patients.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100426"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000165/pdfft?md5=bb9a6504f28f2c39a136326e47d16d30&pid=1-s2.0-S2213048924000165-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103291","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"FLOW CYTOMETRY IN MYELODYSPLASTIC SYNDROME- CORRELATION WITH CYTOMORPHOLOGY RESULTS","authors":"I. Mandac Smoljanovic ,&nbsp;T. Duic ,&nbsp;Z. Siftar ,&nbsp;S. Ostojic Kolonic","doi":"10.1016/j.lrr.2024.100416","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100416","url":null,"abstract":"<div><h3>Introduction</h3><p>In the absence of significant dysplasia in suspected myelodysplastic syndrome (MDS), flow cytometry (FC) analysis helps to distinguish clonal cytopenia. The aim of the study was to analyse the concordance of cytomorphological characteristics and flow cytometry in patients with newly diagnosed MDS in KB Merkur in the period from March 2016 to April 2023.</p></div><div><h3>Methods</h3><p>We analyzed bone marrow FC in 69 patients (38 men, 31 women) who were diagnosed with MDS based on bone marrow morphology.</p></div><div><h3>Results</h3><p>In 57 patients (82%), the morphological diagnosis of MDS was also confirmed in the flow cytometry findings. In 11 (91%) of a total of 12 patients in whom the FC analysis did not prove MDS, no blasts were observed in the cytological findings. The expression of CD34, CD117, CD13 and CD33 (cut off 20%) was analyzed in 57 patients in whom MDS was suspected in the myelogram and FC . CD117 and CD33 did not differ between groups, regardless of MDS subtype. CD34 and CD13 was statistically significantly higher in high-risk MDS than in low-risk MDS. There was no statistically significant difference in the level of hemoglobin and expression of CD antigen, while the level of platelets correlated negatively with the level of CD117, CD13 and CD34. The expression of CD34 positively correlated with the level of C reactive protein.</p></div><div><h3>Conclusions</h3><p>Flow cytometry in MDS is a valuable additional method in discriminating MDS from other forms of cytopenia to improve both diagnosis and prognosis, but there is a need to establish standard techniques in everyday use.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100416"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000062/pdfft?md5=2ae8fd39969090f896f7f77e7f4bf866&pid=1-s2.0-S2213048924000062-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"DDX41 MUTATIONS DEFINE A DISTINCT SUBTYPE OF MYELOID NEOPLASMS.","authors":"H. Makishima","doi":"10.1016/j.lrr.2024.100437","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100437","url":null,"abstract":"<div><h3>Introduction</h3><p>Germline <em>DDX41</em> variants have been implicated in late-onset myeloid neoplasms (MNs). Despite an increasing number of publications, many important features of <em>DDX41</em>-mutated MNs remain unclear.</p></div><div><h3>Methods</h3><p>Here, we enrolled a total of 346 patients with <em>DDX41</em> pathogenic/likely pathogenic (P/LP) germline variants and/or somatic mutations from 9,082 MN patients, together with 525 first-degree relatives of <em>DDX41</em>-mutated and wild-type (WT) patients. We performed a comprehensive characterization of <em>DDX41</em>-mutated MNs.</p></div><div><h3>Results</h3><p>P/LP <em>DDX41</em> germline variants explained ∼80% of known germline predisposition to MNs in adults. These risk variants were 10-fold more enriched in Japanese MN cases (n=4,461) compared to a Japanese general population (n=20,238). This enrichment of <em>DDX41</em> risk alleles was much more prominent in male than female (20.7 vs. 5.0). P/LP <em>DDX41</em> variants conferred a large risk of developing MNs, which was negligible until 40 years old but rapidly increased to 49% by 90 years of age. <em>DDX41</em>-mutated MDS patients rapidly progressed to AML, which was, however, confined to those having truncating variants. Co-mutation patterns at diagnosis and at progression to AML were substantially different between <em>DDX41</em>-mutated and -WT cases, where none of the co-mutations affected clinical outcomes. Even <em>TP53</em> mutations made no exceptions and their dismal effect, including multi-hit allelic status, on survival was almost completely mitigated by the presence of <em>DDX41</em> mutations. Finally, outcomes were not affected by the conventional risk stratifications including the revised/molecular International Prognostic Scoring System (IPSS-R/M).</p></div><div><h3>Conclusions</h3><p>Our findings establish that <em>DDX41</em>-mutated MDS defines a unique subtype of MNs that is distinct from other MNs.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100437"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S221304892400027X/pdfft?md5=9f353ff600f65b73db1fbb5b5da6ac63&pid=1-s2.0-S221304892400027X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"PHASE 3B STUDY DESIGN: COMPARING TREATMENT PREFERENCE BETWEEN ORAL DECITABINE/CEDAZURIDINE AND AZACITIDINE IN PATIENTS WITH MYELODYSPLASTIC SYNDROME, LOW-BLAST ACUTE MYELOID LEUKAEMIA, OR CHRONIC MYELOMONOCYTIC LEUKAEMIA","authors":"A. Enjeti ,&nbsp;C. Fong ,&nbsp;T. Paine ,&nbsp;F. Castaldi ,&nbsp;G. Van Wyk ,&nbsp;R. Walton ,&nbsp;H. Keer","doi":"10.1016/j.lrr.2024.100440","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100440","url":null,"abstract":"<div><h3>Introduction</h3><p>There is uncertainty about how choice of therapy in myelodysplasia (MDS), low-blast count AML (LB-AML) or chronic myelomonocytic leukaemia (CMML) is made by patients, physicians and carers when &gt;1 treatment type is available. This study's primary objective is to compare patients’ treatment preference using the patient ‘treatment preference in myelodysplasia questionnaire’ (pTPMQ). Secondary objectives include evaluation of preference by carers (cTPMQ), and clinicians (mTPMQ).</p></div><div><h3>Methods</h3><p>This phase 3b, open-label, multi-centre study (NCT05883956), with sites in Australia and New Zealand, will compare preference between oral decitabine/cedazuridine (Treatment A) and SC AZA (Treatment B). The study design includes 28 days of screening, four continuous 28-day cycles of study treatment, and a follow-up period with two 28-day cycles of continued therapy. Patients (N=42) will be randomised to two balanced treatment sequences: ABBA or BAAB (Figure 1). Patients will express a preference twice in the study, first after completing Cycle 2, then after completing Cycle 4. This will be assessed via the pTPMQ. Clinician and carer preference will also be assessed.</p></div><div><h3>Results</h3><p>Trial to be activated in Australia and New Zealand, with first patient recruited in December 2023. We will report the primary and secondary objectives. Exploratory objectives including treatment discontinuation rates, quality of life and safety of SC AZA and oral decitabine/cedazuridine will also be reported.</p></div><div><h3>Conclusions</h3><p>This study aims to address an evidence gap in the comparison of patients’, carers’ and clinician's preference between an oral and a parenteral treatment, preference strength, and the reasons for it.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100440"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S221304892400030X/pdfft?md5=d9b2f58e86f8ddbc12e6bbe36dc94e87&pid=1-s2.0-S221304892400030X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103889","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}