Selected and regularly updated scholarly publications of original empirical and theoretical work on thalassaemia and sickle cell disease (SCD), presented in chronological order.
All
α-thalassaemia
Renal Complications
Nurses
Pregnancy
Blood
Bone disease
Sickle Cell Disease
TIF Literature
Liver
Epidemiology
Cardiac complications
Iron Chelation & Overload
Endocrine system
Nutrition
Survival
Hematopoietic Stem Cell Transplantation
Preimplantation Genetic Diagnosis
Gene Therapy and Gene Editing
New Therapeutical Approaches
Prevention
Non-Transfusion Dependent Thalassaemias
Global Burden of Thalassemia by Socio-Demographic Index, 1990–2023: Trends, Disparities, and Future Implications
Sawaira, F., Shahab, S.H., Mal, M. et al. Annals of Hematology. (2026)
EHA Recommendations for Preconceptual and Antenatal Screening and Prenatal Diagnosis for Hemoglobinopathies
de Montalembert, M., Cappellini, M. D., Iolascon, A., de Franceschi, L., Ömur, E., Abi Saad, M. & Rees, D. HemaSphere, 10(6), e70381. (2026)
In Utero Hematopoietic Cell Transplantation in Fetuses With Α-Thalassemia Major: A Phase 1 Clinical Trial
MacKenzie, T. C., Lianoglou, B., Gonzalez-Velez, J., Dvorak, C. C., Kharbanda, S., Herzeg, A. & Vichinsky, E. Blood Advances. (2026)
Transforming The Treatment of Alpha-Thalassemia: A Single-Center Retrospective Study on Hematopoietic Stem Cell Transplantation in Transfusion-Dependent Pediatric Patients
Huang, L., Wei, Z., Yang, G., Liu, L., Zhang, Z., Xiao, H. & Shi, L. Annals of Hematology, 105(4), 157. (2026)
Disease-Related Complications and Risk Factors in Hemoglobin H Disease in a Thai Multicentre Registry
Teawtrakul, N., Songdej, D., Hantaweepant, C., Tantiworawit, A., Hantrakool, S., Sripornsawan, P., et al. Scientific Reports. (2026)
Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease
Gupta, A. O., Sharma, A., Frangoul, H., Kanter, J., Mapara, M. Y., Dalal, J. & BEACON Investigators. N Engl J Med, 394(18), 1824-1835. (2026)
Daily Zinc Supplementation for Infection Prevention in Children With Sickle Cell Anemia: The ZIPS-2 Randomized Clinical Trial
Namazzi, R., Mellencamp, K. A., Bagala, I., Conroy, A. L., Kato, C., Birungi, I. & John, C. C. JAMA. (2026)
Contemporary Global Burden of Sickle Cell Anaemia Under-5 and Under-20: A Systematic Review and Meta-Analysis
Pimenta, K., Edwards, J., Ray, M., Naser, A. M., Price, C., Idassi, O., & Smeltzer, M. British Journal of Haematology. (2026)
Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy
Sharma, A., Kassim, A., Thompson, A., Williams, D. A., Liu, H. D., Boelens, J. J. & Cancio, M. I. Transplantation and cellular therapy. (2026)
Osteoporosis Risk in Alpha and Beta Thalassemia: An Age- and Sex-Specific Retrospective Cohort Study
Hsu, Y. S., Tseng, S. C., Chao, T. F., & Chen, K. H. Archives of Osteoporosis, 21(1), 83. (2026)
Addressing Obstetric and Gynecologic Needs in Thalassemia
Vlachodimitropoulou, E., Baldini, M., Al-Samkari, H., Cappellini, M. D., Coates, T. D., Kuo, K. H., & Musallam, K. M. O&G Open, 3(4), e181. (2026)
Diagnosis and Management of Transfusion-Dependent Thalassemia: Evidence-Based Guidelines From the Pediatric Hematology Oncology Chapter of the Indian Academy of Pediatrics
Radhakrishnan, N., Dewan, P., Chandra, J., Sharma, R., Arora, S., Bhat, S. & Manglani, M. Indian Pediatrics, 1-26. (2026)
Short- and Long-Term Effects of Transfusion in Β-Thalassemia: A Longitudinal Study of Transfusion Efficiency Factors
Theocharaki, K., Barla, I., Delicou, S., Anastasiadi, A. T., Tzounakas, V. L., Rouvela, S. & Antonelou, M. H. Blood Advances, 10(1), 192-207. (2026)
Experiences of Families With Children Diagnosed With Beta-Thalassemia: A Meta-Synthesis of Qualitative Studies
Özkan, İ., Kılıç, K. M., & Taylan, S. Journal of Pediatric Nursing, 89, 301-316. (2026)
Family Empowerment Education Module for Parents of Children With Thalassemia Major: A Randomized Controlled Study on Anxiety, Problem-Solving, and Psychological Resilience
Sülün, A. A., & Kudubeş, A. A. Journal of Pediatric Nursing: Nursing Care of Children and Families, 91, 484-494. (2026)
Transition from Pediatric to Adult Care in Patients with Transfusion-Dependent Beta-Thalassemia in France: A National Study Concerning a Rare Disease
Szepetowski, S., Benoit, A., Berbis, J., Badens, C., Jean, E., de Sainte Marie, B. & Thuret, I. Journal of Clinical Medicine, 15(6), 2203. (2026)
Dapagliflozin in Patients with Homozygous β-Thalassemia and Albuminuria
Tsiotsios, K., Vamvakas, S. S., Labropoulou, V., Lazaris, V., Davoulou, P., Kalavrizioti, D. & Papasotiriou, M. Acta Haematologica. (2026)
Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent β-Thalassemia Treated in the BEYOND Trial
Musallam, K. M., Cappellini, M. D., Kattamis, A., Dyer, M., Hnoosh, A., Eliason, L., & Taher, A. T. European Journal of Haematology, 117(1), 161-173. (2026)
Modelling the Cost-Effectiveness of Non-Invasive Prenatal Testing in the English Sickle Cell and Thalassaemia Screening Pathway
Vardanega, V., Bobrowska, A., Ruban-Fell, B., Doorbar, J. A., Lombardo, S., Seedat, F., et al. Diagnostic and Prognostic Research, 10(1), 17. (2026)
Prevalence of Severe Thalassemia and Performance of Prenatal Screening Tests Among Pregnant Women at Siriraj Thalassemia Center in Thailand
Malasai, K., Chaikledkaew, U., Talungchit, P., Youngkong, S., Udomsinprasert, W., Limwongse, C., & Jittikoon, J. Clinical and Translational Science, 19(2), e70485. (2026)
Systematic Review of Economic Evaluations in Thalassaemia Screening Programmes Globally: Developing Guidance for Low- and Middle-Income (LMIC) Settings
Massey, K., Phommasone, K., Mehta, A., Lee, V. Q. Y., Ashley, E. A., Mayxay, M., & Painter, C. BMJ open, 16(5), e108768. (2026)
Outcomes With Luspatercept in Patients With Β-Thalassemia: A Systematic Review and Metaanalysis
Babiker, N., Abady, E., Shahid, H., Rehman, W. U., Babker, F., Sarhan, M., et al. Annals of Hematology. (2026)
Evaluating Mitapivat for the Treatment of Alpha or Beta Thalassemia
Kattamis, A., Bistas, K., Mitros, V., Agiomavriti Stefanopoulou, E. F., & Delaporta, P. Expert Opinion on Pharmacotherapy. (2026)
Phase 2a Randomized Study to Evaluate Sapablursen in Patients With Non-Transfusion Dependent Β-Thalassemia Intermedia
Taher, A., Diamantidis, M. D., Kattamis, A., Aydinok, Y., Kaplan, Z. S., Dibble, A., et al. Blood Advances-2026019799. (2026)
2026 Update on Clinical Trials in β-Thalassemia
Musallam, K. M., Locatelli, F., Algeri, M., Cappellini, M. D., & Taher, A. T. American Journal of Hematology. (2026)
Long-Term Efficacy and Safety Results of Betibeglogene Autotemcel Gene Therapy for Transfusion-Dependent Β-Thalassemia
Kwiatkowski, J. L., Thompson, A. A., Schneiderman, J., Thuret, I., Kulozik, A. E., Yannaki, E. & Locatelli, F. (2026). Blood, 147(19), 2203-2214. (2026)
Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease
Frangoul, H., de la Fuente, J., Chopra, Y., Meisel, R., Amrolia, P. J., Algeri, M., et al. New England Journal of Medicine. (2026)
Prospective Clinical Validation of Targeted Long-Read Sequencing for Preimplantation Genetic Testing of Α-Thalassaemia
Shi, Q., Fang, H., Zhang, J., Li, N., Xu, C., Liao, Y., et al. Reproductive Biology and Endocrinology. (2026)
Optimizing Outcomes snd Accessibility of Matched Sibling Donor Transplant for Transfusion Dependent Thalassemia in LMICs
Reddy, M., Sridhar, T., Mayur, V., Lathe, G. T., Trivedi, D., Shah, V., et al. Blood Advances. (2026)
Long-Term Survival Rates of Thalassemia Patients Following Hematopoietic Stem Cell Transplantation: A Systematic Review and Meta-Analysis
Adisuhanto, M., Prasetya, A., Cahyadi, A., & Oehadian, A. Hematology, Transfusion and Cell Therapy, 48(4), 106490. (2026)
A Descriptive Preanalytical Survey of Procedures Followed for the Screening of Glucose Dysregulation in Thalassemia Centers: Implications for Clinical Practice and Call for Harmonization
de Sanctis, V., Daar, S., Tzoulis, P., Soliman, A. T., Modeva, I., Kattamis, C. et al. Mediterranean Journal of Hematology and Infectious Diseases, 18(1), e2026035. (2026)
Effects of Vitamin D Deficiency and Supplementation on Myocardial and Hepatic Iron Status and Function in Children with Beta-Thalassemia
Solğun, H. A., & Özay, M. Annals of Hematology, 105(5), 246. (2026)
Endocrinopathies and Growth in a UK Cohort of Children with Transfusion-Dependent Thalassaemia
Sethuraman, C., Yo, W. K., Shone, J., Gevers, E., Kaya, B., & Willemsen, R. H. British Journal of Haematology. (2026)
Pancreatic Iron Overload Is Associated With Early QTc Prolongation Before Myocardial Iron Deposition in Transfusion-Dependent Thalassemia
Meloni, A., Pistoia, L., Ruffo, G. B., Longo, F., Rosso, R., Rossi, V. et al. European Journal of Haematology. (2026)
Neutropenia in Adult Patients with Thalassemia Receiving Deferiprone: A 10-Year Experience From a Large Thalassemia Cohort
Lertvipapath, P., Owatanapanich, W., Uawattanasakul, W., & Kungwankiattichai, S. Blood Research, 61(1), 46. (2026)
Atrial Fibrillation Prevalence and its Management in Aging, Transfusion-Dependent Patients with Thalassemia: The FATHAL Study
DiStefano, V., Gianesin, B., Orecchia, V., Longo, F., Barella, S., Barone, A., et al. Blood Advances, 10(7), 2291-2301. (2026)
MRI-Derived Hepatic Fat and its Clinical Correlates in Non–Transfusion-Dependent Thalassemia: A Cross-Sectional Study
Meloni, A., Pistoia, L., Ricchi, P., Positano, V., Spasiano, A., Longo, F., et al. Blood Cells, Molecules, and Diseases, 102997. (2026)
The Shifting Global Thalassemia Burden and Lessons from Chinese Integrated Control Strategy
Beta‐Thalassemia in Spain: Results From the National Thalassemia Registry and Molecular Analysis of Patients With Transfusion‐Dependent Thalassemia
Villegas, A., Ropero, P., González, F. A., Morado, M., Menor, M., de la Iglesia, S. et al. Journal of Clinical Laboratory Analysis, e70272. (2026)
Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence
Gamaleldin, M. M. A., Abdelhalim, S. M. N. S., & Abraham, I. Thalassemia Reports, 16(2), 7. (2026)
A Multi-Center Clinical Trial of Allogeneic Hematopoietic Stem Cell Transplantation in Transfusion-Dependent Thalassemia
Liu, R., Xiao, H., Qin, C., Hu, J., Luo, J., Chen, H., et al. Nature Communications, 17(1), 3083. (2026)
National Thalassemia Registry: A 30-year Journey of implementing Carrier Screening in Singapore
Zhang, S., et al. The Lancet Regional Health–Western Pacific. (2025)
β-Thalassemia Minor Is Associated With High Rates of Worsening Anemia in Pregnancy
Langer, A. L., Goggins, B. B., Esrick, E. B., Fell, G., Berliner, N., & Economy, K. E. Blood, 145(6), 648-651. (2025)
Impact of Lifetime Anaemia and Iron Control on Outcomes in Β-Thalassaemia: Data from the Longitudinal de-LIGHT Study
Musallam, K. M., Vitrano, A., Inzerillo, A., Di Maggio, R., Barone, R., Giangreco, A., & Maggio, A. British Journal of Haematology, 207(4), 1578-1588. (2025)
Red Cell Specifications for Blood Group Matching in Patients With Haemoglobinopathies: An Updated Systematic Review and Clinical Practice Guideline from the International Collaboration for Transfusion Medicine Guidelines
Wolf, J., Blais‐Normandin, I., Bathla, A., Keshavarz, H., Chou, S. T., Al‐Riyami, A. Z. et al. British journal of haematology, 206(1), 94-108. (2025)
A Guide for the Haemoglobinopathy Nurse
Eleftheriou, A., Angastiniotis, M., & Pelides, K. Thalassaemia International Federation. (2025)
Management of Kidney Disease with Sickle Cell Disease
Abbasi, M., Srivastava, A., & Saraf, S. L. Journal of the American Society of Nephrology, 36(10), 2041-2054. (2025)
Pregnancy and Childbirth in Women With Thalassemia: Past And Present
Demurtas, A., Gianesin, B., Zappu, A., Cassinerio, E., Leoni, S., Ricchi, P. & Origa, R. Haematologica, 111(1), 392. (2025)
The Impact of Various Types of Α-Thalassemia οn Perinatal Complications and Pregnancy Outcomes in Pregnant Women
St-Georges, J., Alnoman, A., Badeghiesh, A., & Baghlaf, H. Archives of Gynecology and Obstetrics, 311(5), 1343-1349. (2025)
Transfusion Strategies in Thalassemia and Sickle Cell Disease SITE-SIMTI-SIdEM Good Practice
Forni, G. L., Vassanelli, A., De Franceschi, L., Marson, P., Lisi, R., Ostuni, A. & Fiorin, F. Blood Transfusion, 23(6), 536. (2025)
Alloimmunization in β-Thalassemia and Sickle Cell Disease in Middle Eastern Countries: A Systemic Review
Al-Allawi, N., Al-Mousawi, M. M., Al Allawi, S., & Ibrahim, K. J. Hemoglobin, 49(2), 126-140. (2025)
Pregnancy, Delivery, and Neonatal Outcomes Among Women With Beta-Thalassemia Major: A Population-Based Study of a Large US Database
St-Georges, J., Alnoman, A., Badeghiesh, A., & Baghlaf, H. Archives of Gynecology and Obstetrics, 311(5), 1343-1349. (2025)
Prevalence and Mortality Trends of Hemoglobinopathies in Italy: A Nationwide Study
Gianesin, B., Musallam, K. M., Barella, S., Casale, M., Forni, G. L. Haematologica. (2025)
Newborn Screening for Sickle Cell Disease in Catalonia between 2015 and 2022: Epidemiology and Impact on Clinical Events
González de Aledo-Castillo, J. M., Argudo-Ramírez, A., Beneitez-Pastor, D., Collado-Gimbert, A., et al. International Journal of Neonatal Screening, 10(4), 69. (2024)
Management of Transfusion-Dependent β-Thalassemia (TDT): Expert Insights and Practical Overview from the Middle East
El-Beshlawy, A., Dewedar, H., Hindawi, S., Alkindi, Taher, A. T. et al. Blood Reviews, 63, 101138. (2024)
Challenges of Iron Chelation in Thalassemic Children
Adramerina, A., & Economou, M. Thalassemia Reports, 14(1), 1-9. (2024)
TIF Literature
All scientific articles by TIF in one place
Renal Findings in Patients with Thalassemia at Abdominal Ultrasound: Should We Still Talk about “Incidentalomas”? Results of a Long-Term Follow-Up
Fatigati, C., Meloni, A., Costantini, S., Spasiano, A., Ascione, F., Cademartiri, F., & Ricchi, P. Diagnostics, 14(18), 2047. (2024)
Iron Chelation Therapy for Children with Transfusion‐Dependent β‐Thalassemia: How Young Is Too Young?
Forni, G. L., Kattamis, A., Kuo, K. H., Maggio, A., Sheth, S., Taher, A. T., & Viprakasit, V. Pediatric Blood & Cancer, e31035. (2024)
Bone Health Impairment in Patients with Hemoglobinopathies: From Biological Bases to New Possible Therapeutic Strategies
Di Paola, A., Marrapodi, M. M., Di Martino, M., Giliberti, G., Di Feo, G., et al. International Journal of Molecular Sciences, 25(5), 2902. (2024)
Early Detection of Renal Complication in Children With Sickle Cell Disease: A Single Center Prospective Study
Alghorayed, R., Alsubayni, B., Hanafy, E., Mustafa, M., Albalawi, N., et al. Cureus, 16(7). (2024)
Hoping for a Normal Life: Decision‐Making on Hematopoietic Stem Cell Transplantation by Patients With a Hemoglobinopathy and Their Caregivers
Mekelenkamp, H., de Vries, M., Saalmink, I., Nur, E., Kerkhoffs, J. L., et al. Pediatric Blood & Cancer, 71(3), e30808. (2024)
Liver Disease in Patients With Transfusion-Dependent β-Thalassemia: The Emerging Role of Metabolism Dysfunction-Associated Steatotic Liver Disease
Fragkou, N., Vlachaki, E., Goulis, I., & Sinakos, E. World Journal of Hepatology, 16(5), 671. (2024)
Global Longitudinal Strain by Cardiac Magnetic Resonance Associated With Cardiac Iron and Complications in β-Thalassemia Major Patients
Meloni, A., Saba, L., Positano, V., Pistoia, L., Campanella, A., et al. International Journal of Cardiology, 132319. (2024)
Revisiting Iron Overload Status and Change Thresholds as Predictors of Mortality in Transfusion-Dependent β-Thalassemia: A 10-year Cohort Study
Musallam, K. M., Barella, S., Origa, R., Ferrero, G. et al. Annals of Hematology, 1-15. (2024)
Global, Regional, and National Burden of Thalassemia, 1990–2021: A Systematic Analysis for the Global Burden of Disease
Tuo, Y., Li, Y., Li, Y., Ma, J., Yang, X., et al. Eclinicalmedicine, 72. (2024)
Growth and Endocrinopathies Among Children with β-Thalassemia Major Treated at Dubai Thalassemia Centre
Almahmoud, R., Hussein, A., Khaja, F. A., Soliman, A. F., Dewedar, H., et al. BMC pediatrics, 24(1), 244. (2024)
Novel Therapeutic Approaches in Thalassemias, Sickle Cell Disease and Other Red Cell Disorders
Pinto, V. M., Mazzi, F., & De Franceschi, L. Blood Journal. (2024)
Health‑Related Quality‑of‑Life Impacts Associated with Transfusion‑Dependent β‑Thalassemia in the USA and UK: A Qualitative Assessment
Drahos, J., Boateng-Kuffour, A., Calvert, M., Levine, L., et. al. The Patient-Patient-Centered Outcomes Research, 1-19. (2024)
Guideline for the Management of Conception and Pregnancy in Thalassaemia Syndromes: A British Society for Haematology Guideline
Shah, F. T., Nicolle, S., Garg, M., Pancham, S., Lieberman, et al. British Journal of Haematology. (2024)
Haemoglobinopathies and Other Rare Anemias in Spain: Ten Years of a Nationwide Registry (REHem-AR)
Marco Sánchez, J. M., Bardón Cancho, E. J., Benéitez, D., et al. Annals of Hematology, 1-13. (2024)
Predicting Factors of Survival Rates Among α-and β-Thalassemia Patients: A Retrospective 10-Year Data Analysis
Iam-arunthai, K., Suwanban, T., Thungthong, P., Chamnanchanunt, S., & Fucharoen, S. Frontiers in Hematology, 3, 1339026. (2024)
An International Learning Collaborative Phase 2 Trial for Haploidentical Bone Marrow Transplant in Sickle Cell Disease
Kassim, A.A., de la Fuente, J., Nur, E., Wilkerson, K.L., Alahmari, A.D. et al. Blood. (2024)
Defining Curative Endpoints for Transfusion-Dependent β-Thalassemia in the Era of Gene Therapy and Gene Editing
Corbacioglu, S., Frangoul, H., Locatelli, F., Hobbs, W. and Walters, M. American Journal of Hematology. 99(3), 422-429. (2024)
Systematic Review and Evidence Gap Assessment of the Clinical, Quality of Life, and Economic Burden of Alpha-Thalassemia
Musallam, K.M., Viprakasit, V., Lombard, L., Gilroy, K., Rane, A., Vinals, et al. eJHaem. (2024)
Magnitude of Bone Disease in Transfusion-Dependent and Non-Transfusion-Dependent β-Thalassemia Patients
Shamoon, R.P., Yassin, A.K., Omar, N., Saeed, M.D., Akram, R., Othman, N.N. and Yassin, A.K. Cureus, 16(3). (2024)
‘Phenoconversion’ in Adult Patients with β-Thalassemia
Musallam, K.M., Barella, S., Origa, R., Ferrero, G.B., Lisi, R., Pasanisi, A., Longo, F., Gianesin, B. and Forni, G.L. American Journal of Hematology. (2024)
Pregnancy Outcomes and Iron Status in β-Thalassemia Major and Intermedia: A Systematic Review and Meta-analysis
Vlachodimitropoulou, E., Mogharbel, H., Kuo, K.H., Ryu, M., Ward, R., Shehata, N. and Malinowski, A.K. Blood Advances. (2024)
A Cross-Sectional, Multicenter, Disease-Specific, Health-Related Quality of Life study in Greek Transfusion Dependent Thalassemia Patients
Klonizakis, P., Roy, N., Papatsouma, I., Mainou, M., Christodoulou, I., Pantelidou, D., Kokkota, S., et al. (2023)
Economic and Clinical Burden of Managing Transfusion-Dependent b-Thalassemia in the United States
Udeze, C., Evans, K.A., Yang, Y., Lillehaugen, T., Manjelievskaia, J., Mujumdar, U., Li, N. and Andemariam, B. Journal of Medical Economics, 26(1), pp.924-932. (2023)
Erythropoiesis in Lower-risk Myelodysplastic Syndromes and Beta Thalassemia
Cappellini, M.D., Taher, A.T., Verma, A., Shah, F. and Hermine, O. Blood Reviews, 59, p.101039. (2023)
Association of Osteoporosis and Sarcopenia with Fracture Risk in Transfusion-Dependent Thalassemia
Thavonlun, S., Houngngam, N., Kingpetch, K., Numkarunarunrote, N., Santisitthanon, P., et al. Scientific Reports, 13(1), p.16413. (2023)
Lung Function Decline in Children with Sickle Cell Disease Treated with Hydroxyurea
Seidl, E., Wilson, D., Odame, I., Kirby‐Allen, M. and Grasemann, H. American Journal of Hematology. (2023)
Splenectomy Significantly Associated with Thrombosis but Not with Pulmonary Hypertension in Patients with Transfusion-Dependent Thalassemia: A Meta-analysis of Observational Studies
Kalamara, T.V., Dodos, K. and Vlachaki, E. Frontiers in Medicine. (2023)
Deferiprone vs Deferoxamine for Transfusional Iron Overload in Sickle Cell Disease and Other Anemias: Pediatric Subgroup Analysis of the Randomized, Open-label FIRST Study
Hamdy, M., El‐Beshlawy, A., Veríssimo, M.P., Kanter, J., Inusa, B., Williams, et al. Pediatric Blood & Cancer. (2023)
Pneumococcal Infections in Children with Sickle Cell Disease Before and After Pneumococcal Conjugate Vaccines
Adamkiewicz, T.V., Yee, M.E., Thomas, S., Tunali, A., Lai, K.W., Omole, F., Lane, P.A. and Yildirim, I. Blood Advances. (2023)
Impact of Imatinib on Reducing the Painful Crisis in Patients with Sickle Cell Disease
Karimi, M., Bahadoram, M., Mafakher, L. and Rastegar, M. Hematology, Transfusion and Cell Therapy. (2023)
Health-Related Quality-of-Life Profile of Pediatric Patients with β-Thalassemia after Hematopoietic Stem Cell Transplantation
Mulas, O., Efficace, F., Orofino, M.G., Piroddi, A. et al. Journal of Clinical Medicine, 12(18), p.6047. (2023)
Modelling the Public Health Impact of Voxelotor in the Management of Sickle Cell Disease in France
Galacteros, F., Ethgen, O. and Beillat, M.. PLOS ONE, 18(9). (2023)
The Danish National Haemoglobinopathy Screening Programme: Report From 16 Years of Screening in a Low-prevalence, Non-endemic Region
Gravholt, E.A.E., Petersen, J., Mottelson, M., Nardo‐Marino, A. et al. British Journal of Haematology. (2023)
Analysis of the Pregnancy Status and Outcomes of Pregnant Women with α-Thalassemia: A Retrospective Clinical Study
Li, Y., Feng, Y., Zhu, Z., Wei, Y., Huang, J., Chen, H., Wei, C., Wei, K. and Pang, L. Research Square. (2023)
How I Τreat Non-Transfusion-Dependent β-Thalassemia
Saliba, A.N., Musallam, K.M. and Taher, A.T., 2023. Blood. (2023)
Pregnancy-related Thromboembolism in Women with Sickle Cell Disease: An Analysis of National Medicaid Data
Agarwal, S., Stanek, J.R., Vesely, S.K., Creary, S.E., et al. American Journal of Hematology. (2023)
Speckle Tracking Echocardiography and Β-Thalassemia Major: A Systematic Review
Patsourakos, D., Aggeli, C., Dimitroglou, Y., Delicou, S., Xydaki, et al. Annals of Hematology, pp.1-22. (2023)
SLN124, a GalNAc Conjugated 19‐mer siRNA Targeting tmprss6, Reduces Plasma Iron and Increases Hepcidin Levels of Healthy Volunteers
Porter, J.B., Scrimgeour, A., Martinez, A., Campion, G.V. et al. American Journal of Hematology. (2023)
Management of the Sickle Cell Trait: An Opinion by Expert Panel Members
Pinto, V.M., De Franceschi, L., Gianesin, B., Gigante, A., et al. Journal of Clinical Medicine, 12(10), p.3441. (2023)
Economic and Clinical Burden of Managing Transfusion-dependent β-Thalassemia in the United States
Udeze, C., Evans, K.A., Yang, Y., Lillehaugen, T., Manjelievskaia, J. et al. Journal of Medical Economics. (2023)
Efficacy and Safety of Early-start Deferiprone in Infants and Young Children with Transfusion-dependent Beta Thalassemia: Evidence for Iron Shuttling to Transferrin in a Randomized, Double-blind, Placebo-controlled, Clinical Trial (START)
Elalfy, M.S., Hamdy, M., Adly, A., Ebeid, F.S., et al. American Journal of Hematology. (2023)
Burden of Aging: Health Outcomes Among Adolescents and Young Adults With Sickle Cell Disease
Howell, K.E., Pugh, N., Longoria, J., Shah, N., Kutlar, A., et al. HemaSphere 7, no. 8 (2023)
Αlpha-thalassemia Genotypes in Vietnam: A Report of 12,030 Pregnant Women and Their Husbands Performing Prenatal Screening for Alpha-thalassemia
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