Impact of Nontransfusion-Dependent Thalassemia on Adult Patients' Health-Related Quality of Life and Work Productivity in the Kingdom of Saudi Arabia and United Arab Emirates
Author(s)
Khaled M. Musallam, PhD, MD1, Maria Domenica Cappellini, MD2, Janet L Kwiatkowski, MD, MSCE3, Vanessa Shih, MS, PharmD4, Amey Rane, BS, MS4, Keely Gilroy, PhD4, Emma Chatterton, BSc, MRes5, Katie Lewis, BSc5, Brianne Kerr, BSc5, Ali T. Taher, MD, PhD, FRCP6.
1Center for Research on Rare Blood Disorders (CR-RBD) and Thalassemia & Sickle Cell Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates, 2Department of Clinical Sciences and Community, University of Milan, Milan, Italy, 3Children’s Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA, 4Agios Pharmaceuticals, Inc., Cambridge, MA, USA, 5Adelphi Real World, Bollington, United Kingdom, 6Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
1Center for Research on Rare Blood Disorders (CR-RBD) and Thalassemia & Sickle Cell Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates, 2Department of Clinical Sciences and Community, University of Milan, Milan, Italy, 3Children’s Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA, 4Agios Pharmaceuticals, Inc., Cambridge, MA, USA, 5Adelphi Real World, Bollington, United Kingdom, 6Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
OBJECTIVES: Understanding of the patient-centric impacts of non-transfusion-dependent thalassemia (NTDT) in the Kingdom of Saudi Arabia (KSA) and the United Arab Emirates (UAE) is limited. This study investigated health-related quality of life and work productivity among patients with NTDT in both countries.
METHODS: Data were collected from the Adelphi Real World Thalassemia Disease Specific Programme™, a non-interventional cross-sectional survey of physicians and their patients from February to November 2024. Eligible patients were aged ≥18 years, with physician-confirmed α- or β-NTDT diagnosis and no involvement in any clinical trial at the time of survey. Patients with a history of gene therapy or hematopoietic stem cell transplantation were excluded. Patients reported demographic characteristics and completed a voluntary survey that captured data from Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue, Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF), and Work Productivity and Activity Impairment (WPAI)-Thalassemia questionnaires. Data were summarized descriptively and compared numerically with published US population norms (PPNs).
RESULTS: Data were collected from 30 patients with NTDT, 15 each from KSA and UAE (66.7% and 53.3% had β-NTDT, respectively). Mean (standard deviation [SD]) ages were 35.2 (6.2) in KSA and 34.0 (4.4) in UAE, and most patients worked full- or part-time (both countries: 80.0%). Mean (SD) FACIT-Fatigue scores were lower in KSA (29.5 [8.6]) and UAE (30.6 [6.6]) versus PPN (43.6 [9.4]), as were PROMIS PF T-scores, 42.9 (5.9) in KSA and 44.3 (4.7) in UAE, versus PPN (59.7 [8.0]). Mean percentage overall impairment score was 53.0% (19.9%) for KSA and 62.3% (20.6%) for UAE, respectively, versus 15.0% (26.0%) PPN.
CONCLUSIONS: In KSA and UAE, adult patients with α- or β-NTDT experience worse fatigue and greater impairment in physical function and work productivity than the general population, highlighting an unmet need to reduce the humanistic burden of NTDT.
METHODS: Data were collected from the Adelphi Real World Thalassemia Disease Specific Programme™, a non-interventional cross-sectional survey of physicians and their patients from February to November 2024. Eligible patients were aged ≥18 years, with physician-confirmed α- or β-NTDT diagnosis and no involvement in any clinical trial at the time of survey. Patients with a history of gene therapy or hematopoietic stem cell transplantation were excluded. Patients reported demographic characteristics and completed a voluntary survey that captured data from Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue, Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF), and Work Productivity and Activity Impairment (WPAI)-Thalassemia questionnaires. Data were summarized descriptively and compared numerically with published US population norms (PPNs).
RESULTS: Data were collected from 30 patients with NTDT, 15 each from KSA and UAE (66.7% and 53.3% had β-NTDT, respectively). Mean (standard deviation [SD]) ages were 35.2 (6.2) in KSA and 34.0 (4.4) in UAE, and most patients worked full- or part-time (both countries: 80.0%). Mean (SD) FACIT-Fatigue scores were lower in KSA (29.5 [8.6]) and UAE (30.6 [6.6]) versus PPN (43.6 [9.4]), as were PROMIS PF T-scores, 42.9 (5.9) in KSA and 44.3 (4.7) in UAE, versus PPN (59.7 [8.0]). Mean percentage overall impairment score was 53.0% (19.9%) for KSA and 62.3% (20.6%) for UAE, respectively, versus 15.0% (26.0%) PPN.
CONCLUSIONS: In KSA and UAE, adult patients with α- or β-NTDT experience worse fatigue and greater impairment in physical function and work productivity than the general population, highlighting an unmet need to reduce the humanistic burden of NTDT.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR131
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)