Center for Research on Rare Blood Disorders (CR-RBD)

Part of Burjeel Holdings

Overview

Our Team

Research Programs

Publications

Books and Guidelines

Congress Abstracts

Clinical Studies

International Collaborators

Recent News

The Center for Research on Rare Blood Disorders (CR-RBD) at Burjeel Medical City (Burjeel Holdings) is fully dedicated to advancing knowledge, addressing evidence gaps, and overcoming unmet needs for patients with rare blood disorders (such as thalassemia, sickle cell disease, hemophilia and other rare bleeding disorders). Our team of scholars and clinicians work together to hand-pick key research questions that reflect diagnostic and management challenges experienced throughout the entire patient journey. We also lead and participate in collaborative research networks at the regional and global level.

Khaled Musallam, MD PhD

Founding Director, CR-RBD
Group Chief Research Officer, Burjeel Holdings

Our Strategy

The overarching strategy of the center is to support better understanding of disease epidemiology and burden, identify risk factors for morbidity, mortality and diminished quality of life, and use such insights to develop novel disease-modifying or curative therapies. This is achieved through the design and conduct of a range of translational and observational studies (including real-world evidence), along with phase 1 to 4 clinical trials of novel agents.

The center is also committed to expanding the literature with expert and systematic reviews on contemporary topics of global interest. We offer enduring research training opportunities for junior scientists and physician-scholars interested in rare blood disorders.

Our Goals

Our ultimate goal is to continually improve outcomes for patients with rare blood disorders and to cement Abu Dhabi and the United Arab Emirates’ rapidly evolving position as a global hub for research and innovation in life sciences.

a. beta-Thalassemia

The beta-thalassemias are a group of rare, inherited disorders of hemoglobin synthesis characterized by ineffective erythropoiesis and anemia of varying severity which can lead to lifelong transfusion dependence. Patients are commonly grouped as having non-transfusion- dependent beta-thalassemia (NTDT) or transfusion-dependent beta-thalassemia (TDT). The beta-thalassemias are particularly common in the Middle East and North Africa region owing to historic association with malaria prevalence, high rate of consanguineous marriages, and ethnic diversity of the population due to a large immigrant population.

For further reading about beta-thalassemia refer to:

Taher AT, Musallam KM, Cappellini MD. N Engl J Med 2021;384(8):727-74.

Our center is leading several research projects focused on patients with NTDT. We have identified that ineffective erythropoiesis and untreated anemia are associated with long-term morbidity and mortality in these patients, contrary to previous beliefs that mild-moderate anemia is well-tolerated and does not require intervention. We have also uncovered specific hemoglobin thresholds that modify the relationship between anemia and outcomes in these patients. These findings have supported the development of several first-in-class novel disease modifying agents that are expected to reshape the natural history of the disease in the next 5 years, including the erythroid maturation agent luspatercept which has been approved in 2023 in Europe, based on data from the BEYOND trial (co-authored by Dr Musallam, with data published in the Lancet Haematology), for use in adults with NTDT, and included in the 2023 version of the Thalassaemia International Federation (TIF) NTDT management guidelines (Dr Musallam being co-Editor).

A more novel agent, the pyruvate kinase activator mitapivat, is being evaluated in the phase 3 ENERGIZE trial, currently ongoing at Burjeel Medical City, for its efficacy and safety in improving anemia in adults with NTDT. Topline data from the trial in January 2024 confirmed that it met its primary objective. At the UAE level, we have initiated a longitudinal cohort (AD-TLC) of beta-thalassemia patients attending various clinical care centers across the Emirate of Abu Dhabi, to better understand persistent disease burden and needs for novel therapies. Our work has also supported understanding of other risk factors and disease outcomes in NTDT, including primary iron overload and hypercoagulability leading to various hepatic, vascular (thrombosis and pulmonary hypertension), and endocrine morbidities.

For patients with TDT, we have also explored the relationship between hemoglobin levels, transfusion burden, and long-term morbidity and mortality outcomes as well as healthcare resource utilization and focus on the development of novel therapies aiming to reduce transfusion requirement in these patients, including mitapivat which is being evaluated in the phase 3 ENERGIZE-T trial at Burjeel Medical City. Transfusion therapy leads to accumulation of iron in critical organs like the heart and liver, and subsequent end-organ damage. The prospect of patients with TDT has been completely transformed with the development of modern techniques for iron overload assessment (e.g., R2 and T2* MRI) and oral iron chelation therapy. However, adherence, intolerance, and efficacy limitations of current iron chelators continue to be concerns in a large subset of TDT patients. Our center is also involved in the development of novel iron chelators in this patient population. We have also conducted large cohort studies to revisit iron overload levels that are associated with highest risk of morbidity and mortality to inform decision making and chelation strategies.

b. alpha-Thalassemia

Patients with alpha-thalassemia have been less commonly studied than those with beta-thalassemia. Our center has conducted various systematic literature reviews to identify data gaps, focused on disease epidemiology, clinical burden, quality of life and economic burden. This work provided important insights that are currently being analyzed to provide direction for the development of longitudinal cohort studies and disease registries. We are also establishing the first global cohort on alpha-thalassemia to fill evidence gaps on risk factors and outcomes.

For further reading about alpha-thalassemia refer to:

Musallam KM, et al. Αlpha-thalassemia: a practical overview. Blood Rev 2024;64:101165.

Peer-reviewed journals

Musallam KM, Barella S, Origa R, Ferrero GB, Lisi R, Pasanisi A, Longo F, Gianesin B, Forni GL; on behalf of the Webthal^® project. Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent β-thalassemia patients who remained on the same monotherapy over 10 years.
Blood Cells Mol Dis 2024;107:102859.
Access
Musallam KM, Barella S, Origa R, Ferrero GB, Lisi R, Pasanisi A, Longo F, Gianesin B, Forni GL; on behalf of the Webthal^® project. Revisiting iron overload status and change thresholds as predictors of mortality in transfusion-dependent β-thalassemia: a 10-year cohort study.
Ann Hematol 2024;103(7):2283-2297.
Access
Musallam KM, Viprakasit V, Lombard L, Gilroy K, Rane A, Vinals L, Tam C, Rizzo M, Coates TD. Systematic review and evidence gap assessment of the clinical, quality of life, and economic burden of alpha-thalassemia.
EJHaem 2024;5:541-547.
Access
Bizri M, Koleilat R, Akiki N, Dergham R, Mihailescu AM, Bou-Fakhredin R, Musallam KM, Taher AT. Quality of life, mood disorders, and cognitive impairment in adults with β-thalassemia.
Blood Rev 2024;65:101181.
Access **

Musallam KM, Cappellini MD, Coates TD, Kuo KHM, Al-Samkari H, Sheth S, Viprakasit V, Taher AT. Αlpha-thalassemia: a practical overview.
Blood Rev 2024;64:101165.
Access **
Musallam KM, Barella S, Origa R, Ferrero GB, Lisi R, Pasanisi A, Longo F, Gianesin B, Forni GL; on behalf of the Webthal^® project. ‘Phenoconversion’ in adult patients with β-thalassemia.
Am J Hematol 2024;99(3):490-493.
Access **
Musallam KM, Barella S, Origa R, Ferrero GB, Lisi R, Pasanisi A, Longo F, Gianesin B, Forni GL; on behalf of the Webthal^® project. Pretransfusion hemoglobin level and mortality in adults with transfusion-dependent β-thalassemia.
Blood 2024;143(10):930-932.
Access **
Musallam KM, Sheth S, Cappellini MD, Kattamis A, Kuo KHM, Taher AT. Luspatercept for transfusion-dependent β-thalassemia: time to get real.
Ther Adv Hematol 2023;14: 20406207231195594.
Access
Saliba AN, Musallam KM, Taher AT. How I treat non-transfusion-dependent β-thalassemia.
Blood 2023;142(11):949-960.
Access **
Musallam KM, Lombard L, Nutr M, Kistler KD, Arregui M, Gilroy KS, Chamberlain C, Zagadailov E, Ruiz K, Taher AT. Epidemiology of Clinically Significant Forms of Alpha- and Beta-Thalassemia: A Global Map of Evidence and Gaps.
Am J Hematol 2023;98(9):1436-1451.
Access **
Musallam KM, Taher AT, Kattamis A, Kuo KHM, Sheth S, Cappellini MD. Profile of luspatercept in the treatment of anemia in adults with non-transfusion-dependent β-thalassemia (NTDT): design, development and potential place in therapy.
Drug Des Devel Ther 2023;17:1583-1591.
Access
Musallam KM, Ahmed W, Almarshoodi MO, Trad O, Al Zein N, Siddique S, Shekary N, Daboul S, Hashmi S. Establishment of the Abu Dhabi Emirate Thalassemia Longitudinal Cohort (AD-TLC): Leveraging the Power of Observation.
Ann Hematol 2023;102(8):2277-2278.
Access
Taher AT, Musallam KM, Viprakasit V, Kattamis A, Lord-Bessen J, Yucel A, Guo S, Pelligra C, Shields AL, Shetty JK, Miteva D, Bueno LM, Cappellini MD. Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia.
BMJ Open 2023;13:e066683.
Access
Forni GL, Gianesin B, Musallam KM (co-second author), Longo F, Rosso R, Lisi R, Gamberini MR, Pinto VM, Graziadei G, Vitucci A, Bonetti F, Musto P, Piga A, Cappellini MD, Borgna-Pignatti C on behalf of the Webthal^® project. Overall and complication-free survival in a large cohort of patients with β-thalassemia major followed over 50 years.
Am J Hematol 2023;98(3):381-387.
Access **
Musallam KM, Taher AT, Cappellini MD, Hermine O, Kuo KHM, Sheth S, Viprakasit V, Porter JB. Untreated anemia in non-transfusion-dependent β-thalassemia (NTDT): Time to sound the alarm.
HemaSphere 2022;6(12):e806.
Access **
Vitrano A, Musallam KM (co-first author), Meloni A, Karimi M, Daar S, Ricchi P, Costantini S, Vlachaki E, Di Marco V, El-Beshlawy A, Hajipour M, Hussain Ansari S, Filosa A, Ceci A, Singer ST, Naserullah ZA, Pepe A, Cademartiri F, Pollina SA, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A; International Working Group on Thalassemia (IWG-THAL). Development of a Thalassemia International Prognostic Scoring System (TIPSS).
Blood Cells Mol Dis 2023;99(2023):102710.
Access
Musallam KM, Taher AT, Cappellini MD. Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia.
Cell Rep Med 2022;3(10):1-2.
Access **
Musallam KM, Cappellini MD, Daar S, El-Beshlawy A, Taher AT. Magnitude of cumulative iron overload correlates with the severity of anemia in untreated non-transfusion-dependent β-thalassemia.
Ann Hematol 2023;102(2):467-469.
Access
Taher AT, Cappellini MD, Kattamis A, Voskaridou E, Perrotta S, Piga A, Filosa A, Porter JB, Coates TD, Forni GL, Thompson AA, Tartaglione I, Musallam KM, Backstrom JT, Esposito O, Giuseppi AC, Kuo W-L, Miteva D, Lord-Bessen J, Yucel A, Zinger T, Shetty JK, Viprakasit V, on behalf of the BEYOND Investigators. Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial.
Lancet Haematol 2022;9(10):e733-e744.
Access **
Derchi G, Musallam KM (co-first author), Pinto VM, Graziadei G, Giuditta M, Barella S, Origa R, Casu G, Pasanisi A, Longo F, Casale M, Miceli R, Merella P, Gianesin B, Ameri P, Tartaglione I, Perrotta S, Piga A, Cappellini MD, Forni GL on behalf of the Webthal^® project. Tricuspid-valve regurgitant jet velocity as a risk factor for death in β-thalassemia.
Haematologica 2022;107(7):1714-1718.
Access **
Vitrano A, Musallam KM (co-first author), Meloni A, Pollina SA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E, Taher AT, Sankaran VG, Maggio A; International Working Group on Thalassemia (IWG-THAL). Random forest clustering identifies three subgroups of β-thalassemia with distinct clinical severity.
Thalassemia Rep 2022;12(1):14-23.
Access
Pinto VM, Musallam KM (co-first author), Derchi G, Graziadei G, Giuditta M, Origa R, Barella S, Casu G, Pasanisi A, Longo F, Casale M, Miceli R, Merella R, Tartaglione I, Piga A, Cappellini MD, Gianesin B, Forni GL on behalf of the Webthal^® project. Mortality in β-thalassemia patients with confirmed pulmonary arterial hypertension on right heart catheterization.
Blood 2022;139(13):2080-2083.
Access **
Hodroj MH, El Hasbani G, Al-Shamsi HO, Samaha H, Musallam KM, Taher AT. Clinical burden of hemophilia in older adults: beyond bleeding risk.
Blood Rev 2022;53:100912.
Access **
Musallam KM, Vitrano A, Meloni A, Pollina SA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E, Taher AT, Sankaran VG, Maggio A; International Working Group on Thalassemia (IWG-THAL). Risk of mortality from anemia and iron overload in non-transfusion-dependent β-thalassemia.
Am J Hematol 2022:97(2):E78-E80.
Access **
Musallam KM, Vitrano A, Meloni A, Pollina SA, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Karimi M, El-Beshlawy A, Hajipour M, Bonifazi F, Vichinsky E, Taher AT, Sankaran VG, Maggio A; International Working Group on Thalassemia (IWG-THAL). Primary HBB gene mutation severity and long-term outcomes in a global cohort of β-thalassemia.
Br J Haematol 2022;196(2):414-423.
Access **
Tang C-H, Furnback W, Wang BCM, Tang J, Tang D, Lu M-Y, Huang VWH, Musallam KM. Relationship between transfusion burden, healthcare resource utilization, and complications in patients with beta-thalassemia in Taiwan: a real-world analysis.
Transfusion 2021;61(10):2906-2917.
Access
El Hasbani G, Musallam KM, Uthman I, Cappellini MD, Taher AT. Thalassemia and autoimmune diseases: absence of evidence or evidence of absence?
Blood Rev 2022;52:100874.
Access **
Musallam KM, Bou-Fakhredin R, Cappellini MD, Taher AT. 2021 Update on Clinical Trials in β-Thalassemia.
Am J Hematol 2021;96(11):1518-1531.
Access **
Musallam KM, Vitrano A, Meloni A, Pollina SA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A; International Working Group on Thalassemia (IWG-THAL). Survival and causes of death in 2033 patients with non-transfusion-dependent β-thalassemia (NTDT).
Haematologica 2021;106(9):2489-2492.
Access **

** Indicates publication in a Tier 1 (first quartile) journal according to Clarivate JCR impact factor at the time of acceptance.

Taher A, Al-Samkari H, Aydinok Y, Bartolucci P, Besser M, Dahlin JL, Estepp JH, Gheuens S, Gilroy KS, Glenthøj A, Goh AS, Iyer V, Kattamis A, Loggetto SR, Morris S, Musallam KM, Osman K, Ricchi P, Salido-Fiérrez E, Sheth S, Tai F, Tevich H, Uhlig K, Urbstonaitis R, Viprakasit V, Cappellini MD, Kuo KHM. ENERGIZE: A Global Phase 3 Study of Mitapivat Demonstrating Efficacy and Safety in Adults with Alpha- or Beta- Non—Transfusion-Dependent Thalassemia.
European Hematology Association Congress, Madrid-Spain; June 13-16, 2024. abs. S104. Hemasphere 2024;8(S1):11-12. [PLENARY ORAL PRESENTATION]
Kuo KHM, Al-Samkari H, Aydinok Y, Besser M, Boscoe AN, De Luna G, Estepp JH, Gheuens S, Gilroy KS, Glenthøj A, Goh AS, Kattamis A, Loggetto SR, Morris S, Musallam KM, Osman K, Ricchi P, Salido-Fiérrez E, Sheth S, Tai F, Uhlig K, Viprakasit V, Cappellini MD, Taher A. Improvements in Fatigue and 6-Minute Walk Test in Adults with Alpha- or Beta-Non—Transfusion-Dependent Thalassemia: The Phase 3 ENERGIZE Trial of Mitapivat.
European Hematology Association Congress, Madrid-Spain; June 13-16, 2024. abs. P1529. Hemasphere 2024;8(S1):2830-2831.

Kuo KHM, Kattamis A, Taher AT, Motta I, Cappellini MD, Musallam KM, Bueno LM, Perin M, Medlin LF, Martin-Regueira P, Lai Y, Ugidos M, Sheth S. Characterizing Patterns of Transfusion Burden (TB) Reduction in Patients (PTS) with Transfusion-Dependent (TD) Βeta-Thalassemia Treated With Luspatercept in the BELIEVE Trial.
European Hematology Association Congress, Madrid-Spain; June 13-16, 2024. abs. P1522. Hemasphere 2024;8(S1):2816-2817.
Kattamis A, Musallam KM, Perrotta S, Aydinok Y, Ferrero GB, Coates TD, Grabowska O, Bluemmert I, Simcock M, Bueno LM, Viprakasit V. Safety Data from the Dose-Finding Cohorts: A Phase 2a Study of Luspatercept in Pediatric Patients With Βeta-Thalassemia. Characterizing Patterns of Transfusion Burden (TB) Reduction in Patients (PTS) with Transfusion-Dependent (TD) Βeta-Thalassemia Treated With Luspatercept in the BELIEVE Trial.
European Hematology Association Congress, Madrid-Spain; June 13-16, 2024. abs. P1516. Hemasphere 2024;8(S1):2804-2805.
Taher AT, Viprakasit V, Kattamis A, Perrotta, Ricchi P, Porter JB, Coates TD, Forni GL, Musallam KM, Esposito O, Giuseppi AC, Kuo W-L, Reverte M, Wei R, Bueno LM, Cappellini MD. Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent β-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial.
65^th American Society of Hematology Annual Meeting and Exposition, San Diego, CA-USA; December 9-12, 2023. abs. 3847. Blood (ASH Annual Meeting Abstracts) 2023;142(Suppl 1):3847.
Musallam KM, Taher AT, Kattamis A, Cappellini MD, Viprakasit V, Lord-Bessen J, Glassberg M, Pelligra C, Guo S, Martin P, Medlin LF, Bueno LM, Kuo KHM. Durable Symptom Improvement for Patients with Non-Transfusion Dependent Thalassemia Treated with Luspatercept: Patient-Reported Outcomes from the BEYOND Study.
65^th American Society of Hematology Annual Meeting and Exposition, San Diego, CA-USA; December 9-12, 2023. abs 2474. Blood (ASH Annual Meeting Abstracts) 2023;142(Suppl 1):2474.
Taher AT, Viprakasit V, Cappellini MD, Piga AG, Porter JB, Coates TD, Musallam KM, Forni GL, Shetty J, Bosilkovska-Weisskopf M, Wei R, Kuo W-L, Kattamis A. Long-term erythroid response data from non-transfusion-dependent patients with β-thalassemia receiving luspatercept in the BEYOND Trial. Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent β-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial.
The 16th TIF International Conference on Thalassaemia & Haemoglobinopathies & the 18th TIF International Conference for Patients and Parents, Kuala Lumpur-Malaysia; November 3-5, 2023.
Viprakasit V, Coates TD, Musallam KM, Buerki JV, Patturajan M, Holot N, Aydinok Y. A phase 2a study evaluating the safety and pharmacokinetics (PK) of luspatercept in pediatric patients with transfusion-dependent β-thalassemia (TDT).
The 16th TIF International Conference on Thalassaemia & Haemoglobinopathies & the 18th TIF International Conference for Patients and Parents, Kuala Lumpur-Malaysia; November 3-5, 2023.
Taher AT, Viprakasit V, Cappellini MD, Piga AG, Porter JB, Coates TD, Musallam KM, Forni GL, Shetty J, Bosilkovska-Weisskopf M, Wei R, Kuo W-L, Kattamis A. Long-term erythroid response data from patients (pts) with non-transfusion-dependent beta-thalassemia (NTDT) receiving luspatercept in the BEYOND trial.
European Hematology Association Congress, Frankfurt-Germany; June 8-11, 2023. abs. S273. Hemasphere 2023;7(Suppl):e45673ef. [ORAL PRESENTATION]
Taher AT, Viprakasit V, Cappellini MD, Piga AG, Porter JB, Coates TD, Musallam KM, Forni GL, Shetty J, Bosilkovska-Weisskopf M, Wei R, Kuo W-L, Kattamis A. Erythroid Response in Patients with Non-Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Long-Term Data from the BEYOND Trial.
64^th American Society of Hematology Annual Meeting and Exposition, New Orleans, LA-USA; December 10-13, 2022. abs. 3669. Blood (ASH Annual Meeting Abstracts) 2022;140(Suppl 1):8210-8212.
Musallam KM, Taher AT, Porter, Kattamis A, Glassberg MB, Bueno L, Shetty JK, Lersch F, Rosettani B, Cappellini MD. A Closer Look at Changes in Hemoglobin Levels in Patients with Non-Transfusion Dependent β-Thalassemia Treated with Luspatercept: Post Hoc Analysis of the Phase 2 BEYOND Trial.
64^th American Society of Hematology Annual Meeting and Exposition, New Orleans, LA-USA; December 10-13, 2022. abs. 2349. Blood (ASH Annual Meeting Abstracts) 2022;140 (Suppl 1):5358-5359.
Musallam KM, Lombard L, Gilroy K, Vinals L, Rizzo M. Characterizing the Clinical, Health-Related Quality of Life and Economic Burden of Alpha-Thalassemia: A Systematic Literature Review and Evidence Gaps Assessment.
64^th American Society of Hematology Annual Meeting and Exposition, New Orleans, LA-USA; December 10-13, 2022. abs. 1036. Blood (ASH Annual Meeting Abstracts) 2022;140 (Suppl 1):2491-2493.
Musallam KM, Taher AT, Lombard L, Nutr M, Gilroy K, Lewis K, Mellor J, Golden K, Cappellini MD. Real-World Evidence Characterizing the Burden of Disease in Alpha and Beta Thalassemia Patients in the USA: an Interim Analysis.
ASCAT-EHA-BSH Sickle Cell Disease Conference 2022, London-United Kingdom; October 20-22, 2022. abs. 5610611. Hemasphere 2023;7 (Suppl):27.
Musallam KM, Vinals L, Gilroy K, Rizzo M, Lombard L. Systematic literature review of health-related quality of life burden in patients across the spectrum of thalassemia.
ASCAT-EHA-BSH Sickle Cell Disease Conference 2022, London-United Kingdom; October 20-22, 2022. abs. 5589252. Hemasphere 2023;7 (Suppl):12-13. [ORAL PRESENTATION]
Musallam KM, Vinals L, Gilroy K, Rizzo M, Lombard L. Systematic literature review of health-related quality of life burden in patients across the spectrum of thalassemia.
Eleventh Cooley’s Anemia Symposium, NY-USA; October 17-20, 2022.
Cario H, Viprakasit V, Coates TD, Musallam KM, Buerki JV, Patturajan M, Holot N, Aydinok Y. Luspatercept in Pediatric Patients (pts) With Transfusion-Dependent β-Thalassemia (TDT): Design and Eligibility Criteria of a Phase 2a Study Evaluating Safety and Pharmacokinetics (PK) of Luspatercept in Children.
Annual Meeting of the German, Austrian and Swiss Associations of Hematology and Medical Oncology, Vienna-Austria; October 7-10, 2022. abs. V578. [ORAL PRESENTATION]
Musallam KM, Vinals L, Gilroy K, Rizzo M, Lombard L. Systematic literature review of health-related quality of life burden in patients across the spectrum of thalassemia.
European Hematology Association Congress, Vienna-Austria; June 9-12, 2022. abs. PB2233. Hemasphere 2022;6(Suppl):2103-2104.
Viprakasit V, Coates TD, Musallam KM, Buerki JV, Patturajan M, Holot N, Aydinok Y. A Phase 2a Study Evaluating the Safety and Pharmacokinetics of Luspatercept in Pediatric Patients with Transfusion-Dependent β-Thalassemia (TDT).
63^rd American Society of Hematology Annual Meeting and Exposition, Atlanta, GA-USA; December 11-14, 2021. abs. 4161. Blood (ASH Annual Meeting Abstracts) 2021;138(Suppl 1):4161.

Genetic and hematologic determinants of outcomes in β-thalassemia: the Abu Dhabi Emirate Thalassemia Longitudinal Cohort (AD-TLC)

Principal Investigator	Dr. Khaled Musallam
Design	Observational cohort
Population	Beta-thalassemia, all ages
Status	Active — recruiting
Local approval codes	BH/REC/037/22, DOH/CVDC/2022/1808, DOH/CVDC/2024/393
Sponsor(s)	Department of Health (DoH) - Abu Dhabi (UAE)
Links	N/A

An open-label, dose-escalation, dose-finding, and proof-of-concept trial of SP-420 in subjects with transfusion-dependent β-thalassemia

Principal Investigator	Dr. Khaled Musallam
Design	Phase 2, open-label
Population	Transfusion-dependent beta-thalassemia, adults
Status	Active – recruiting
Local approval codes	BH/REC/030/22, DOH/CVDC/2022/1627, DOH/CVDC/2023/48, DOH/CVDC/2023/1635, DOH/CVDC/2023/1984
Sponsor(s)	Pharmacosmos A/S (Denmark)
Links	clinicaltrials.gov identifier (NCT05693909)

A phase 3, double-blind, randomized, placebo-controlled, multicenter study evaluating the efficacy and safety of mitapivat in subjects with transfusion-dependent alpha — or beta-thalassemia (ENERGIZE-T)

Principal Investigator	Dr. Khaled Musallam
Design	Phase 3, randomized, double-blind, placebo-controlled
Population	Transfusion-dependent thalassemia (TDT), adults
Status	Active — not recruiting
Local approval codes	BH/REC/008/2022, DOH/CVDC/2022/529, DOH/CVDC/2023/477, DOH/CVDC/2023/587, DOH/ADHRTC/2024/1130, DOH/ADHRTC/2024/2020
Sponsor(s)	Agios Pharmaceuticals (USA)
Links	clinicaltrials.gov identifier (NCT04770779)

A phase 3, double-blind, randomized, placebo-controlled, multicenter study evaluating the efficacy and safety of mitapivat in subjects with non-transfusion-dependent alpha- or beta-thalassemia (ENERGIZE)

Principal Investigator	Dr. Khaled Musallam
Design	Phase 3, randomized, double-blind, placebo-controlled
Population	Non-Transfusion-dependent thalassemia (NTDT), adults
Status	Active – not recruiting
Local approval codes	BH/REC/007/2022, DOH/CVDC/2022/528, DOH/CVDC/2023/478, DOH/CVDC/2023/586, DOH/ADHRTC/2024/1131, DOH/ADHRTC/2024/2021
Sponsor(s)	Agios Pharmaceuticals (USA)
Links	clinicaltrials.gov identifier (NCT04770753)

If you are interested in joining or learning more about our clinical studies, please refer to the contact us section.

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