Clinical Burden and Transfusion-Related Outcomes in Transfusion-Dependent Thalassemia 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: To characterize clinical burden and transfusion-related outcomes in patients with transfusion-dependent thalassemia (TDT) in the Kingdom of Saudi Arabia (KSA) and United Arab Emirates (UAE).
METHODS: Data were collected from the Adelphi Real World Thalassemia Disease Specific Programme™, a cross-sectional survey of physicians and their patients from February-November 2024. Patients: ≥18 years; physician-confirmed α- or β-TDT diagnosis; no involvement in any clinical trial at time of survey. Patients with history of gene therapy/hematopoietic stem cell transplantation were excluded. Physicians reported patient demographics and clinical characteristics, including complications (concomitant conditions ever experienced), current/historical transfusion-related outcomes, and current treatment patterns. Data were summarized descriptively.
RESULTS: Data were collected from 122 patients, 60 from KSA (α-TDT: n=17; β-TDT: n=43); 62 from UAE (α-TDT: n=16; β-TDT: n=46), with mean (standard deviation) ages of 24.8 (5.7) and 27.1 (6.5) years, respectively. In KSA, the most frequent complications ever experienced were iron overload (per physician’s assessment; 81.7%), splenomegaly (53.3%), moderate-severe hepatic iron overload (definition: >7.0 mg/g or >57µmol/g; 51.7%), hepatomegaly (45.0%), and infections (35.0%); in UAE, they were iron overload (100.0%), moderate-severe hepatic iron overload (69.4%), splenomegaly (58.1%), osteoporosis (40.3%), and hepatomegaly (37.1%). Overall, 100.0% and 90.3% of patients with TDT in KSA and UAE experienced acute or long-term complications due to transfusions; the most common was iron overload, 93.3% in KSA and 87.1% in UAE. Blood supply issues (past 12 months) affected 66.7% of patients in KSA and 54.8% in UAE, while 70.0% and 77.4%, respectively, experienced hospitalization (past 12 months) due to thalassemia and related complications. Most patients in KSA and UAE received supportive treatment, including chelation therapies (97.5%), folic acid (90.2%), and calcium (56.6%).
CONCLUSIONS: In KSA and UAE, patients with TDT experience clinical burden, transfusion-related complications, and blood supply issues, highlighting an unmet need for novel therapies to reduce disease, transfusion, and treatment burden.
METHODS: Data were collected from the Adelphi Real World Thalassemia Disease Specific Programme™, a cross-sectional survey of physicians and their patients from February-November 2024. Patients: ≥18 years; physician-confirmed α- or β-TDT diagnosis; no involvement in any clinical trial at time of survey. Patients with history of gene therapy/hematopoietic stem cell transplantation were excluded. Physicians reported patient demographics and clinical characteristics, including complications (concomitant conditions ever experienced), current/historical transfusion-related outcomes, and current treatment patterns. Data were summarized descriptively.
RESULTS: Data were collected from 122 patients, 60 from KSA (α-TDT: n=17; β-TDT: n=43); 62 from UAE (α-TDT: n=16; β-TDT: n=46), with mean (standard deviation) ages of 24.8 (5.7) and 27.1 (6.5) years, respectively. In KSA, the most frequent complications ever experienced were iron overload (per physician’s assessment; 81.7%), splenomegaly (53.3%), moderate-severe hepatic iron overload (definition: >7.0 mg/g or >57µmol/g; 51.7%), hepatomegaly (45.0%), and infections (35.0%); in UAE, they were iron overload (100.0%), moderate-severe hepatic iron overload (69.4%), splenomegaly (58.1%), osteoporosis (40.3%), and hepatomegaly (37.1%). Overall, 100.0% and 90.3% of patients with TDT in KSA and UAE experienced acute or long-term complications due to transfusions; the most common was iron overload, 93.3% in KSA and 87.1% in UAE. Blood supply issues (past 12 months) affected 66.7% of patients in KSA and 54.8% in UAE, while 70.0% and 77.4%, respectively, experienced hospitalization (past 12 months) due to thalassemia and related complications. Most patients in KSA and UAE received supportive treatment, including chelation therapies (97.5%), folic acid (90.2%), and calcium (56.6%).
CONCLUSIONS: In KSA and UAE, patients with TDT experience clinical burden, transfusion-related complications, and blood supply issues, highlighting an unmet need for novel therapies to reduce disease, transfusion, and treatment burden.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO43
Topic
Clinical Outcomes
Topic Subcategory
Clinician Reported Outcomes
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)