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4583 Improvement in Quality of Life in MDS Patients Who Become Transfusion Independent after Treatment

Program: Oral and Poster Abstracts
Session: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
Research, MDS, Clinical Research, Chronic Myeloid Malignancies, Patient-reported outcomes, Diseases, Real-world evidence, Myeloid Malignancies
Monday, December 9, 2024, 6:00 PM-8:00 PM

Bo Wan, MD1, Shabbir MH Alibhai, MD, MSc2*, Lisa Chodirker, MD FRCPC3*, Lee Mozessohn, MD4, Michelle Geddes, MD5, Nancy Zhu, MD, FRCPC6*, Amy Trottier, MD, MSc, FRCPC7, Eve St-Hilaire, MD, FRCP8*, Nicholas Finn, BSc, MD9*, Brian Leber, MD10, Dina Khalaf, MSc, MBBS11, Grace Christou, MD, MSc, FRCPC12*, Mitchell Sabloff, MSc, MD, FRCPC13, Heather A Leitch, MD, PhD14, April Shamy, MD15, Karen Yee, MD, FRCPC16*, John M. Storring, MDCM, FRCPC17, Thomas J. Nevill, MD, FRCPC18, Brett L. Houston, MD, PhD19, Mohamed Elemary, MD, MSc, PhD20*, Robert Delage, MD, MSc21, Anne Parmentier, BA22*, Mohammad Siddiqui23*, Alexandre Mamedov, PhD4*, Liying Zhang, PhD4* and Rena Buckstein, MD, FRCPC3

1University of British Columbia, Vancouver, BC, Canada
2Department of Supportive Care, Princess Margaret Cancer Centre - University Health Network, Toronto, Canada
3Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, ON, Canada
4Sunnybrook Health Sciences Centre, Toronto, ON, Canada
5Tom Baker Cancer Centre, University of Calgary, Calgary, AB, Canada
6Clinical Assistant Professor Division of Hematology, Department of Medicine, University of Alberta, Edmonton, AB, Canada
7Hematology, Queen Elizabeth II Health Sciences Centre, Halifax, NS, Canada
8Hematology/Oncology, Dr. Georges-L-Dumont University Centre, Moncton, NB, CAN
9Hematology/oncology, Dr. Georges-L-Dumont University Centre, Moncton, CAN
10Division of Hematology, Juravinski Cancer Center, Hamilton, ON, Canada
11Department of Oncology, McMaster University, Hamilton, ON, Canada
12Division of Hematology, The Ottawa Hospital, Ottawa, ON, Canada
13Ottawa Hospital Research Institute, Ottawa, ON, Canada
14Hematology, St. Paul's Hospital, University of British Columbia, Vancouver, BC, Canada
15Jewish General Hospital, McGill University, Montreal, QC, CAN
16Princess Margaret Cancer Centre, University of Toronto, Toronto, Canada
17McGill University Health Centre, Montreal, QC, Canada
18Division of Hematology, Department of Medicine, University of British Columbia, Vancouver, Canada
19University of Manitoba, Winnipeg, MB, CAN
20Provincial Hematology and Blood and Marrow Transplant Program, Saskatchewan Cancer Agency, University of Saskatchewan, Saskatoon, SK, Canada
21Quebec CML-MPN Research Group, Montreal, QC, Canada
22Sunnybrook Health Sciences, Odette Cancer Center, Toronto, ON, Canada
23Hematology Clinical Trials, Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Canada

Introduction: Patients with myelodysplastic syndromes (MDS) generally experience inferior quality of life (QOL), in part due to transfusion dependent-anemia and. Many studies have shown that patients who are transfusion dependent (TD) typically have worse QOL compared to patients who are transfusion independent (TI), but the impact of achieving TI once TD on patient QOL is uncertain. MDS-CAN is a prospective multicentre Canadian registry that captures longitudinal clinical and QOL ‘real-world’ data in patients with MDS. We used the MDS-CAN registry to provide additional insight into the association between changes in transfusion status (i.e., from TD to TI, or from TI to TD) or stability of transfusion state (TI and TD) with overall survival (OS) and QOL.

Methods: We conducted a retrospective cohort analysis of red blood cell (RBC) transfusion status over time and examined its impact on OS and patient reported outcomes in patients prospectively enrolled in the MDS-CAN registry (NCT02537990). In brief, enrollment started in 2006 and included patients who were ≥ 18 years old with a diagnosis of MDS, MDS/MPN, or low blast count AML (20-30%). The four transfusion groups were defined as TD to TI (Group A), TI to TD (Group B), and maintaining TD (Group C) or TI (Group D).

We obtained data on demographics, disease, function, frailty, laboratory parameters, as well as longitudinal data on treatment, transfusions, and patient-related health outcomes using validated instruments assessing QOL. The earliest QOL score obtained after initiation of treatment was considered the baseline for each patient. In group A and B, this was compared to the QOL score obtained at the first instance after a change in transfusion status. To be consistent, the median time to change in transfusion status in group A and B was used to select the comparator QOL time point in groups C and D. Wilcoxon rank-sum or Kruskal-Wallis nonparametric test, Chi-square or Fisher exact test were applied to compare continuous or categorical demographic and treatment characteristics, as appropriate. A general linear mixed model was used to identify significant associations between QOL scores and the 4 transfusion health states. Kaplan-Meier overall survival (OS) curves from diagnosis were conducted in each of the four groups (A, B, C, and D), in patients treated with hypomethylating agent (HMA), and in patients treated with erythropoietin stimulating agonist (ESA).

Results:

Of 1120 patients included from the MDS-CAN registry, 759 were treated with either an HMA, lenalidomide or ESA. Longitudinal QOL analysis of the 656 patients who received treatment and provided serial QOL data included 54, 151, 126, and 326 patients in groups A-D, respectively. Overall QOL significantly improved in Group A, with trends towards improved component scores in physical and social function. In Group B, there were significant decreases from baseline to the first QOL after TD across multiple QOL measures on several instruments including worse global QOL, worse fatigue as well as worse physical, role, and social functioning. Group C had significantly more fatigue; group D had significant but milder deteriorations across global QOL, fatigue, physical and role functioning.

Survival curves for all registry patients (n=1120) and the HMA-treated cohort (n=427) demonstrated superior OS in patients in group D. This was followed by groups A and B with similar OS to each other and was the lowest in group C (p<0.001 for both curves). These differences in OS were observed after 36 months of follow up. There were no differences in survival between the four groups among those who received ESA.

Conclusion:

Patients with MDS receiving treatment who achieved TI had improvements in patient-reported overall QOL but not in all individual metrics. Loss of TI was associated with a greater negative impact on overall QOL, with significance extending across multiple domains of physical, role, and emotional function. Among patients who remained TI, there was a trend of gradually reduced QOL over time. Patients who remained TI had superior OS, and patients who remained TD had inferior OS. There was intermediate OS in patients who had a change in transfusion status of either direction.

Disclosures: Mozessohn: Abbvie: Honoraria. Geddes: Pfizer: Consultancy, Honoraria; Servier: Consultancy, Honoraria; Astellas: Consultancy, Honoraria; Taiho: Consultancy, Honoraria, Research Funding; AbbVie: Consultancy, Honoraria, Research Funding; Amgen: Research Funding; Syros: Research Funding; Novartis: Consultancy, Honoraria; BMS: Consultancy, Honoraria, Research Funding; GSK: Consultancy, Honoraria. Zhu: Bristol Myers Squibb: Consultancy, Membership on an entity's Board of Directors or advisory committees; Janssen Pharmaceuticals,: Membership on an entity's Board of Directors or advisory committees; Taiho: Consultancy, Membership on an entity's Board of Directors or advisory committees. St-Hilaire: BMS: Membership on an entity's Board of Directors or advisory committees; Taiho: Membership on an entity's Board of Directors or advisory committees. Finn: Takeda: Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees. Leber: Abbvie: Consultancy, Research Funding, Speakers Bureau; Treadwell: Research Funding, Speakers Bureau; Alexion/GSK: Research Funding, Speakers Bureau; SOBI: Research Funding, Speakers Bureau; Amgen: Research Funding, Speakers Bureau; Astex: Research Funding, Speakers Bureau; Paladin: Research Funding, Speakers Bureau; Pfizer: Research Funding, Speakers Bureau; Astellas: Research Funding, Speakers Bureau; Jazz: Research Funding, Speakers Bureau; Novartis: Research Funding, Speakers Bureau; Otsuka: Research Funding, Speakers Bureau; Celgene: Research Funding, Speakers Bureau; BMS: Research Funding, Speakers Bureau; Roche: Research Funding, Speakers Bureau; Servier: Research Funding, Speakers Bureau; Janssen: Research Funding, Speakers Bureau. Khalaf: Novartis: Honoraria; Pfizer: Honoraria; Paladin: Honoraria. Christou: AbbVie Corporation: Honoraria; Bristol Myers Squibb: Honoraria; TAIHO pharma: Honoraria. Sabloff: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Abbvie: Honoraria, Membership on an entity's Board of Directors or advisory committees; Astellas: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bristol Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Jazz: Research Funding. Leitch: BeiGene: Honoraria; Janssen: Honoraria; AstraZeneca: Honoraria. Shamy: Astra Zeneca: Honoraria; BMS: Membership on an entity's Board of Directors or advisory committees. Yee: Astex: Other: research support; Forma Therapeutics: Other: research support; F. Hoffmann-La Roche: Other: research support; Genentech: Other: research support; Geron: Other: research support; Gilead Sciences: Other: research support; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: research support; Bristol Myers Squibb/Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; GSK: Membership on an entity's Board of Directors or advisory committees; Jazz: Membership on an entity's Board of Directors or advisory committees; Shattuck Labs: Membership on an entity's Board of Directors or advisory committees; Taiho Pharma: Membership on an entity's Board of Directors or advisory committees; Taiho Pharma: Honoraria. Storring: Abbvie: Membership on an entity's Board of Directors or advisory committees, Other: Clinical Trails, Speakers Bureau; Astellas: Membership on an entity's Board of Directors or advisory committees, Other: Clinical Trails, Speakers Bureau; BMS/Celgene: Membership on an entity's Board of Directors or advisory committees, Other: Clinical Trails, Speakers Bureau; Novartis: Membership on an entity's Board of Directors or advisory committees, Other: Clinical Trails, Speakers Bureau; Pfizer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Taiho: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Membership on an entity's Board of Directors or advisory committees; Jazz: Membership on an entity's Board of Directors or advisory committees; Teva: Membership on an entity's Board of Directors or advisory committees; Syndax: Other: Clinical Trails. Buckstein: BMS: Honoraria, Research Funding; Abbvie: Honoraria; Taiho: Honoraria, Research Funding; Keros: Other: Advisory Board.

*signifies non-member of ASH