-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

3800 Myelodysplastic Neoplasms Patients’ and Caregivers’ Unmet Needs and Their Impact on Their Quality of Life and Symptoms

Program: Oral and Poster Abstracts
Session: 908. Outcomes Research: Myeloid Malignancies: Poster II
Hematology Disease Topics & Pathways:
Research, Clinical Practice (Health Services and Quality), Clinical Research, Supportive Care, Patient-reported outcomes
Sunday, December 8, 2024, 6:00 PM-8:00 PM

Elena Crisa, MD1*, Daniela Cilloni, MD2*, Marta Riva3*, Enrico Balleari, MD4*, Daniela Barraco, MD5*, Lorenza Borin, MD6*, Michela Calmasini7*, Anna Calvisi, MD8*, Isabella Capodanno, MD9*, Matteo Giovanni Della Porta, MD10*, Elisa Diral, MD11*, Bruno Fattizzo, MD12, Susanna Fenu, MD13*, Stefania Paolini, MD, PhD14*, Carlo Finelli, MD14*, Claudio Fozza, MD15*, Chiara Frairia, MD16*, Valentina Giai, MD, PhD17*, Mauro Turrini, MD18*, Maria Antonia Isoni, MD19*, Federico Itri, MD20*, Luca Maurillo, MD21*, Alfredo Molteni, MD22*, Giuseppe Alberto Palumbo, MD23*, Anna Maria Pelizzari, MD24*, Federica Pilo, MD25*, Antonella Poloni, PhD26*, Costanza Bosi, MD27*, Grazia Sanpaolo, MD28*, Rosaria Sancetta, MD29*, Cristina Amato, SN30*, Valeria Santini, MD30, Maria Teresa Voso, MD31, Sam Salek32*, Tatyana Ionova33*, Annamaria Nosari, MD34* and Esther Natalie Oliva, MD35

1Candiolo Cancer Institute, FPO-IRCCS, Candiolo, Italy
2Department of Clinical and Biological Sciences, University of Turin, Orbassano, Italy
3Department of Hematology and Oncology, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy
4IRCCS Policlinico Ospedale San Martino, Genova, Italy
5Dipartimento di Ematologia - ASST Sette Laghi, Ospedale di Circolo e Fondazione Macchi, Varese, Italy
6Hematology, Irccs San Gerardo di Monza, Monza, ITA
7Associazione Italiana Pazienti Sindromi Mielodisplastica AIPASIM, milano, Italy
8SC Ematologia,CTMO e Laboratorio specialistico, Ospedale S Francesco, Nuoro, Italy
9Hematology Unit, Azienda Unità Sanitaria Locale-IRCCS, Reggio Emilia, Italy
10IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy
11Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy
12Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy
13Hematology and Stem Cell Transplant, St. Giovanni Addolorata Hospital, Rome, Italy
14IRCCS Azienda Ospedaliero-Universitaria di Bologna – Institute of Hematology “Seràgnoli", Bologna, Italy
15Hematology Unit – Azienda Ospedaliera Universitaria of Sassari, University of Sassari, Sassari, Italy
16Hematology, AOU Città della Salute e della Scienza di Torino, Torino, Italy
17Hematology, AOU Città della Salute e della Scienza di Torino, Turin, Italy
18Hematology Unit, Division of Hematology, Valduce Hospital, Como, Como, Italy
19Istituto di Ematologia, Università di Sassari, Sassari, Italy, sassari, Italy
20Dipartimento di Scienze Cliniche e Biologiche, AOU San Luigi Gonzaga, Orbassano, Italy
21Department of Onco-Hematology, Fondazione Policlinico Tor Vergata, Rome, Italy
22Ospedale Niguarda Ca' Granda, Milano, ITA
23Department of Scienze Mediche, Chirurgiche e Tecnologie Avanzate “G.F. Ingrassia”, University of Catania, Catania, Italy
24ASST Spedali Civili Di, Brescia, Italy
25Hematology and BMT Unit, A.Businco hospital, Cagliari, Italy
26Hematology Unit, Department of Clinical and Molecular Sciences, Università Politecnica delle Marche, Ancona, Ancona, Italy
27Ematologia e Centro Trapianti, Ospedale Guglielmo di saliceto, Piacenza, Italy
28Hematology Unit, IRCCS Casa Sollievo Della Sofferenza, San Giovanni Rotondo, Italy
29Hematology Unit, Dell'Angelo Hospital, Venezia-Mestre, Italy
30MDS Unit, Hematology, DMSC, AOU Careggi, University of Florence, Florence, Italy
31Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
32School of Life and Medical Sciences, University of Hertfordshire, Hatfield, United Kingdom
33St. Petersburg State University Hospital, Saint-Petersburg, Russian Federation
34Associazione Italiana Pazienti Sindromi Mielodisplastica AIPASIM, Milano, ITA
35Hematology Unit, Grande Ospedale Metropolitano BMM, Reggio Calabria, Reggio Calabria, Italy

Background and aims. Myelodysplastic neoplasms (MDS) prevail in the elderly and are generally incurable. Thus, maintaining/improving quality of life (QoL) is one of the main goals of treatment. There are many factors related to MDS and unmet needs that may impact QoL of patients and their caregivers. The aim of this study was therefore to explore the impact of MDS on patients and caregivers, their preferences and unmet needs in Italy.

Methods. The survey (online and paper versions) was promoted by the Associazione Italiana Pazienti Sindromi Mielodisplastica (AIPaSiM) advocacy group. Sixty items relevant to patients and 20 relevant to family caregivers were identified by hematologists and patients. The patient-reported outcome measures (PROMs), QOL-E and HM-PRO, were included in the survey. Lower scores in QOL-E and higher scores in HM-PRO represent worse outcomes.

Results. From June 2022 to May 2023, 259 patients of median age 73 (range 27-98) years, 56% female and 71 family caregivers (80% females and 94% family members) completed the survey. MDS duration was over 5 years in 28% of cases. Seventy percent of patients were receiving treatment: 64% for lower risk and 36% for higher risk MDS. Only 15% of patients felt that treatment impacted negatively on everyday life. Seventy-seven percent of patients were retired. MDS affected work life of 20% of patients, leading to changes in job type or working hours or to retirement/activity stop. Distance from the treating center was far for 20% and 48% needed a caregiver to accompany them to hospital. For 44%, travelling to hospital was claimed distressful in terms of physical and economic burden and impacted on family members and work. Comparing PROMs of these patients to the remaining 56%, distress was associated with significantly worse median HM-PRO physical (PB, 50 vs 21, p<0.001), emotional (EB, 54 vs 32, p<0.001), social (SB, 33 vs 0, p=0.001), eating and drinking habits (ED, 34 vs 19, p<0.001), Part A (39 vs 15, p<0.001) and Part B symptom score (SS, 20 vs 9, p<0.001) and QOL-E physical (Ph, 50 vs 62, p<0.001), functional (Fun, 22 vs 89, p<0.001), social (Soc, 25 vs 75, p<0.001), fatigue (Fat, 64 vs 81, p<0.001), general (Gen, 47 vs 72, p=0.003), MDS specific (MDSS, 44 vs 74, p<0.001) and treatment-outcome index scores (TOI, 38 vs 70, p<0.001).

Forty-two percent of patients received regular red blood cell transfusions. The increasing frequency of transfusions (<2/4 units/month, 34%); 2-4 units/month, 53%; >4 units, 13%) was associated with worse HM-PB (p=0.042), SB (p=0.038) and part A scores (p=0.029) and QOL-E Soc (p=0.012), Fat (p= 0.034), MDSS (p=0.002) and Gen scores (p=0.049). Only 2% received transfusions at home; however, 38% would prefer being transfused at home, 77% of which experienced distress reaching the hospital, even though only 20% were far from the center of care. Such patients, in fact, had worse PROMs. Waiting time in hospital was unacceptable for 40% of patients and was associated with worse HM-PRO PB (p=0.014), EB (p<0.001), SB (p=0.025), Part A (p<0.001) and SS scores (p=0.002), and QOL-E Fun (p=0.004), Soc (p=0.012), Fat (p=0.001), MDSS (p=0.001), Gen (p=0.003) and TOI scores (p=0.003). Telemedicine communication was preferred in 54% of cases, particularly if reaching hospital was distressful (62% yes vs 45% no, p=0.035)

Other unmet needs were comprehension of diagnosis for 15% and treatment options for 21%, and access to new drugs for 35%. Thirty-six percent of patients had a family caregiver of median age 56 (28-83) years and 16% of patients felt to be a burden for their family. The caregiver’s work was affected in 34% leading to a job change in 29% of them. On a scale 0-10 on the impact on work-life, family caregivers scored median 4 (interquartile range 2-6).

Conclusions

This Italian study has highlighted the issues related to patients with MDS and their caregivers. More time should be dedicated to the communication of the diagnosis, prognosis and treatment options. Furthermore, for both patients and caregivers, there is an economic burden and distress in attending and waiting in hospital. For patients, transfusion frequency is correlated with worse QoL. These data highlight the need of treatments that reduce transfusion burden, hospital visits and allow patients to be treated at home. Identifying patients’ and caregivers’ unmet needs and their impact on QoL and symptoms can provide optimized patient care and guide hematologists and governance choices.

Disclosures: Balleari: ASL1 Imperiese -Italy: Ended employment in the past 24 months. Della Porta: Bristol Myers Squibb: Consultancy. Fattizzo: Novartis: Consultancy; Samsung: Speakers Bureau; Sobi: Speakers Bureau; Agios: Research Funding; Zenas BioPharma: Research Funding; Roche: Consultancy, Other: travel to congress; Alexion: Consultancy; Janssen: Consultancy. Finelli: Celgene BMS: Consultancy; Novartis: Consultancy; Takeda: Consultancy. Fozza: Sanofi: Research Funding; Amgen: Research Funding; BMS: Research Funding; Soby: Consultancy. Giai: Sobi: Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Other: All authors received support for third-party writing assistance, furnished by Akshaya Srinivasan, PhD, CMPP, of Nucleus Global, an Inizio company, and funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland.; Incyte: 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; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Alexion: Honoraria, Membership on an entity's Board of Directors or advisory committees. Palumbo: Novartis: Consultancy, Honoraria; Morphosys: Consultancy, Honoraria; GSK: Consultancy, Honoraria; Incyte,: Consultancy, Honoraria; Bristol-Myers Squibb: Consultancy, Honoraria; AstraZeneca: Consultancy, Honoraria; AOP: Consultancy, Honoraria; AbbVie: Consultancy, Honoraria. Pelizzari: Grifols International S.A.: Other: Travel & Lodging; Bristol Myers Squibb S.r.l. Italy: Other: Travel & Lodging. Santini: Servier: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees; BMS: Membership on an entity's Board of Directors or advisory committees; Abbvie: Membership on an entity's Board of Directors or advisory committees, Other: Travel support; Jazz: Membership on an entity's Board of Directors or advisory committees, Other: Travel support; Keros: Membership on an entity's Board of Directors or advisory committees; Geron: Membership on an entity's Board of Directors or advisory committees; Syros: Membership on an entity's Board of Directors or advisory committees; Ascentage: Membership on an entity's Board of Directors or advisory committees; CTI: Membership on an entity's Board of Directors or advisory committees; Curis: Membership on an entity's Board of Directors or advisory committees. Voso: Celgene/BMS: Other: Research support, Advisory Board, Speakers Bureau; Novartis: Other: Research support, Speakers Bureau; Astra Zeneca: Speakers Bureau; Syros: Other: Advisory Board; Abbvie: Speakers Bureau; Jazz: Other: Advisory Board, Speakers Bureau; Astellas: Speakers Bureau. Ionova: Sobi, Novartis: Research Funding. Oliva: Alexion: Consultancy, Honoraria, Speakers Bureau; Ryvu: Consultancy, Honoraria, Patents & Royalties; Sobi: Consultancy, Honoraria, Speakers Bureau; Janssen: Speakers Bureau; Daiichi Sankyo: Consultancy, Honoraria; Halia Therapeutics: Patents & Royalties; Novartis: Consultancy, Honoraria, Speakers Bureau; Amgen: Consultancy, Honoraria, Speakers Bureau; Bristol Myers Squibb: Consultancy, Honoraria, Speakers Bureau; Grande Ospedale Metropolitano Bianchi Melacrino Morelli: Current Employment.

*signifies non-member of ASH