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406 Intraindividual Comparison of Recognition of Symptom Burden in MPN between Patient- and Physician-Reported Assessment and Its Impact on Survival – an Analysis By the German Study Group for MPN (GSG-MPN)

Program: Oral and Poster Abstracts
Type: Oral
Session: 908. Outcomes Research: Myeloid Malignancies: Patient Reported Outcomes and their Association with Clinical Outcomes in Patients with Myeloid Malignancies
Hematology Disease Topics & Pathways:
MPN, Clinical Practice (Health Services and Quality), Chronic Myeloid Malignancies, Diseases, Myeloid Malignancies
Saturday, December 7, 2024: 4:45 PM

Kirsi Manz, PhD1,2*, Florian H. Heidel, MD3, Steffen Koschmieder, MD4,5, Rudolf Schlag, MD6*, Joerg Lipke, MD7*, Frank Stegelmann, MD8*, Martin Griesshammer, Prof. Dr. med.9, Martine Klausmann10*, Carl Crodel11*, Andreas Hochhaus, MD12, Holger Schulz13*, Joachim R. Goethert, MD14, Haifa Kathrin Al-Ali, MD; PhD15,16*, Heiko Becker, MD17*, Andreas Reiter, MD18, Gernot Beutel, MD3*, Kim Kricheldorf19,20*, Tim H. H. Brummendorf, MD5,21, Wolfgang Hoffmann, MD1*, Konstanze Döhner, MD22 and Susanne Isfort23*

1Institute for Community Medicine, University Medicine Greifswald, Greifswald, Germany
2Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School (MHH), Hannover, AL, Germany
3Department of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
4Center for Integrated Oncology Aachen Bonn Cologne Duesseldorf (CIO ABCD), Aachen, Germany
5Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Faculty of Medicine, University Hospital RWTH Aachen, Aachen, Germany
6Practice for Hematology and Oncology Wuerzburg, Wuerzburg, Germany
7Practice for Hematology and Oncology, Dortmund, Cologne, Germany
8University Hospital of Ulm, Department of Internal Medicine III, Ulm, Germany
9University Clinic for Hematology, Oncology, Hemostasis and Palliative Care, Johannes Wesling Medical Center, University of Bochum, Minden, Germany
10Gemeinschaftspraxis Aschaffenburg, Aschaffenburg, Germany
11Department of Hematology and Oncology, Comprehensive Cancer Center Central Germany – Campus Jena, Jena University Hospital, Jena, Germany
12Hematology and Oncology, Comprehensive Cancer Center Central Germany – Campus Jena, Klinik für Innere Medizin II, Jena, Germany
13Oncological Practice, Frechen, DEU
14Department of Hematology and Stem Cell Transplantation, West German Cancer Center (WTZ), University Hospital Essen, Essen, Germany
15Krukenberg Cancer Center Halle, Universitätsmedizin Halle, Halle (saale), Sachsony-anhalt, Germany
16Universitätsmedizin Halle, Halle, DEU
17Department of Hematology, Oncology and Stem Cell Transplantation, Faculty of Medicine, University of Freiburg, Freiburg, Germany
18Department of Hematology and Oncology, University Medical Centre Mannheim, Mannheim, Germany
19Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, Faculty of Medicine, University Hospital RWTH Aachen, Aachen, Germany
20Center for Integrated Oncology - Aachen Bonn Cologne Duesseldorf (CIO-ABCD), Aachen, Germany
21Center for Integrated Oncology Aachen-Bonn-Cologne-Düsseldorf (CIOABCD), Aachen, Germany
22Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany
23Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School (MHH), Hannover, Germany

Myeloproliferative neoplasms (MPN) are associated with a variety of symptoms that severely impact patients' quality of life and ability to perform daily activities. Recent studies reported on differences in the perception of physician- versus patient-reported symptom burden. So far, direct comparisons of patient- and physician-reported scales are lacking. Moreover, impact of symptom burden on overall survival had so far shown inconclusive results.

The German Study Group MPN Bioregistry is an observational study of BCR::ABL1-negative MPN patients with > 70 participating centers, including academic and community hospitals as well as office-based hematologists. Here, a prospective analysis on symptom burden of 3,979 MPN patients was conducted to intra-individually compare reports of physicians (present or absent, as documented in the medical record) versus patients (as rated on a scale of 1 to 10 using a questionnaire), collected at the same time (73% within a week). Cohen’s kappa was calculated to assess the degree of agreement between patient and physician reports. Adjusted Cox models were fitted to investigate the effect of symptom burden on survival.

77% of all GSG-MPN registry patients at the time of data extraction had at least one common patient-physician symptom assessment at baseline or any follow-up visit. Characteristics of patients in our cohort were comparable to those in the whole registry. Overall, 93% reported the presence of at least one symptom at baseline. Severe symptom burden (symptom score ≥ 7) was reported in 38% of the patients (range depending on phenotype 34%-44%). Fatigue was the most prevalent symptom reported by both patients (82-85%) and physicians (26% (ET, MPN-U) to 36% (MF; p < 0.001)). Night sweats, pain and pruritus were the second most common symptoms, followed by weight loss and fever. Comparability of baseline symptom prevalence between physician and patients´ assessment was low. Even for objectively measurable symptoms, discrepancies became obvious: unintentional weight loss in the past 6 months was reported by 26% (706/2687) of patients but only 6% of physicians (169/2687). For all symptoms, patients reported a higher prevalence than physicians. Concordance was low and improved slightly for symptoms with a severity score of ³ 4. Concordance was similar for patients with and without cytoreductive therapy, and symptom prevalence was not lower in those who received cytoreductive therapy. No trend towards better physician symptom recognition over time was recorded. Of note, the presence of any symptom (physician-rated) and any severe symptom (patient-reported) at baseline was associated with a higher hazard of death in multivariable models adjusted for age, sex, and disease duration (adj. HR = 1.6 (CI: 1.3, 1.9), p < 0.001 and adj. HR = 1.5 (CI: 1.2, 1.9), p < 0.001, respectively). Among the six symptoms, severe fatigue at baseline and severe weight loss at baseline were both associated with increased hazard of death, with adj. HR = 1.8 (CI: 1.4, 2.3), p < 0.001 and adj. HR = 3.5 (CI: 2.4, 5.0), p < 0.001, respectively. Symptom persistence in general did not result in an increased hazard of death, while, specifically, persistent weight loss led to a higher chance of a fatal outcome (adj. HR = 6.3 (CI: 2.3, 17.0), p < 0.001).

Taken together, this analysis on > 3900 MPN patients reports on significant discrepancies in symptom recognition using an intra-individual assessment between patient-reported and physician-reported data. Physicians reported symptom less frequent compared to patients. Physician- and patient-reported symptoms were an independent risk factor for short-term death and should therefore be carefully assessed in clinical routine.

Disclosures: Heidel: BMS/Celgene, Novartis, CTI: Research Funding; BMS/Celgene, AOP, Novartis, CTI, Janssen, Abbvie, GSK, Merck, Kartos, Telios: Consultancy. Koschmieder: Member of EHA Guidelines Committee, Chairman Hemostasis Working Party of DGHO, Co-Speaker of German Study Group for MPN (GSG-MPN) and GSG-MPN bioregistry: Membership on an entity's Board of Directors or advisory committees; RWTH Aachen University: Patents & Royalties: Patent filed on BET inhibitors; Alexion, Novartis, BMS, Incyte / Ariad, AOP Pharma, Baxalta, CTI, Pfizer, Sanofi, Celgene, Shire, Janssen, Geron, Kartos, Protagonist, Sierra Oncology, Glaxo-Smith Kline, Imago Biosciences, AbbVie, iOMEDICO, MSD: Speakers Bureau; Novartis Foundation, BMS, Novartis, AOP Pharma, Janssen/Geron: Research Funding; Pfizer, Incyte / Ariad, Novartis, AOP Pharma, BMS, Celgene, Geron, Janssen, CTI, Roche, Baxalta, Sanofi, MPN Hub, Protagonist, Sierra Oncology, Glaxo-Smith Kline, AbbVie, PharmaEssentia, MSD: Consultancy; Novartis, BMS, Pfizer, Incyte, Ariad, Shire, Roche, AOP Pharma, Janssen, Geron, Celgene, Kartos, Abbvie, iOMEDICO, MSD: Honoraria. Stegelmann: Pfizer: Consultancy, Honoraria, Speakers Bureau; Novartis: Consultancy, Honoraria, Speakers Bureau; MorphoSys: Consultancy, Honoraria, Speakers Bureau; Incyte: Consultancy, Honoraria, Speakers Bureau; GSK: Consultancy, Honoraria, Speakers Bureau; BMS: Consultancy, Honoraria, Speakers Bureau; Abbvie: Consultancy, Honoraria, Speakers Bureau. Griesshammer: AOP Orphan, Novartis, BMS, AbbVie, Pfizer, Roche, Janssen, Gilead, AstraZeneca, Sierra, Lilly, GSK: Consultancy, Honoraria. Crodel: BMS: Consultancy, Honoraria; GSK: Consultancy, Honoraria; BeiGene: Consultancy; AstraZeneca: Consultancy, Honoraria; Novartis: Honoraria, Other: Travel support; AOP Orphan: Honoraria; Synlab: Honoraria. Hochhaus: Novartis: Honoraria, Other: Advisory Board, Research Funding; Terns: Honoraria, Other: Advisory Board; Enliven: Honoraria, Other: Advisory Board. Goethert: Incyte: Consultancy, Honoraria, Other: Travel support; Pharma&: Honoraria; AOP Orphan Pharmaceuticals: Consultancy, Honoraria, Other: Travel support; Blueprint Medicines: Consultancy, Honoraria; GSK: Consultancy, Honoraria; Novartis: Consultancy, Honoraria, Other: Travel support; Pfizer: Consultancy, Honoraria; BMS: Consultancy, Honoraria, Other: Travel support; Imago Biosciences: Consultancy; porteros biostructures: Consultancy; CTI Biopharma: Consultancy; Abbvie: Consultancy. Al-Ali: AOP: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria, Other: Travel grant; Blueprint: Honoraria; Otsuka: Consultancy, Honoraria; Stemline: Honoraria; BMS: Consultancy, Research Funding; GSK: Consultancy, Honoraria; MSD: Honoraria; Blueprint: Consultancy, Honoraria; Alexion: Other: Travel grant; Incyte: Research Funding; Novartis: Consultancy, Honoraria. Becker: Abbvie: Honoraria; Novartis: Honoraria; Pierre Fabre Pharma: Honoraria; BMS: Honoraria; GSK: Honoraria; Lilly: Honoraria; MSD: Honoraria; Servier: Honoraria. Reiter: Novartis: Consultancy, Honoraria, Other: Grants (institution) , Research Funding; Blueprint Medicines Corporation: Consultancy, Other: Grants (institution) , Research Funding; Cogent Therapeutics LLC: Research Funding; Abbvie: Research Funding; AOP: Consultancy, Honoraria, Other: travel grants, Research Funding; Astra Zeneca: Research Funding; BMS: Research Funding; GSK: Consultancy, Honoraria, Other: travel expense, Research Funding; Incyte: Research Funding. Brummendorf: Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Patents & Royalties: Combination of Imatinib with hypusination inhibitors, Research Funding; Merck: Honoraria; Ariad: Consultancy, Honoraria; Repeat Dx: Consultancy, Research Funding; Pfizer: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Gilead: Consultancy, Honoraria; Roche: Honoraria. Hoffmann: Pfizer: Honoraria. Döhner: AbbVie, AOP Health, Janssen, Jazz, Novartis, Bristol Myers Squibb, Celegne: Consultancy, Honoraria; Novartis, AbbVie, Astellas, Bristol Myers Squibb, Celegne, Jazz Pharmaceuticals, Kronos Bio, Servier: Research Funding. Isfort: Hexal: Other: Travel support; Alexion: Other: Travel support; AOP Orphan: Honoraria, Other: Travel support; Incyte: Honoraria; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel support; Silence Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel support; GSK: Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Other: Travel support; Mundipharma: Other: Travel support.

*signifies non-member of ASH