Selected and regularly updated scholarly publications of original empirical and theoretical work on thalassaemia and sickle cell disease (SCD), presented in chronological order.
All
Sickle Cell Disease
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
α-thalassaemia
Renal Complications
Nurses
Pregnancy
Blood
Bone disease
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)
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)
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. (2023)
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
Cuong, T.D., Ngoc, N.P., Van Anh, T., Linh, D.A., Anh, N.T., et al. Medical Journal of Indonesia. (2023)
Low-dose Warfarin May Be a Life-saving Treatment Regimen in Sickle Cell Diseases
Helvaci, M.R. et al. World Family Medicine. (2023)
Epidemiology of Clinically Significant Forms of alpha- and beta-Thalassemia: A Global Map of Evidence and Gaps
Musallam, M. K., Lombard, L., Kistler, K., Arregui, M., Gilroy, K. et al. American Journal of Hematology. (2023)
Examining Mental Health, Education, Employment, and Pain in Sickle Cell Disease
Venous Thromboembolism Prophylaxis in Sickle Cell Disease: A Multicenter Cohort Study of Adolescent Inpatients
Davila, J., Stanek, J., & O'Brien, S. H. Blood Advances, 7(9), pp.1762-1768. (2023)
Health-related Quality of Life in Patients with β-Thalassemia: Data from the Phase 3 BELIEVE Trial of Luspatercept
Cappellini, M.D., Taher, A.T., Piga, A., Shah, F., Voskaridou, E., Viprakasit, V., Porter, J.B. et al. European Journal of Haematology. (2023)
Longitudinal study of ICET-A on Glucose Tolerance, Insulin Sensitivity and β-cell Secretion in Eleven β-Thalassemia Major Patients with Mild Iron Overload
De Sanctis, V., Soliman, A. T., Daar, S., Tzoulis, P., Di Maio, S., & Kattamis, C. Acta Bio Medica: Atenei Parmensis. (2023)
Treating Chronic Pain in Sickle Cell Disease – The Need for a Biopsychosocial Model
Childerhose, J. E., Cronin, R. M., Klatt, M. D., & Schamess, A. New England Journal of Medicine. (2023)
Management of Luspatercept Therapy in Patients with Transfusion-dependent β-Thalassaemia
Sheth, S., Taher, A. T., Coates, T. D., Kattamis, A., & Cappellini, M. D. British Journal of Haematology. (2023)
Deferiprone for Transfusional Iron Overload in Sickle Cell Disease and Other Anemias: Open-label Study of Up to 3 Years
Elalfy, M.S., Hamdy, M., El-Beshlawy, A., Ebeid, F.S., Badr, M., Kanter, J., Inusa, B., et al. Blood Advances, 7(4), pp.611-619. (2023)
Multi-Parametric Cardiac Magnetic Resonance for Prediction of Heart Failure Death in Thalassemia Major
Meloni, A., Pistoia, L., Gamberini, M.R., Cuccia, L., Lisi, et al. Diagnostics, 13(5). (2023)
A Novel, Effective, and Efficient Strategy for Treating Sickle Cell Vaso-occlusive Events in the Infusion Center Setting
Reduction in Vaso-Occlusive Events Following Hematopoietic Stem Cell Transplantation in Patients with Sickle Cell Disease
Leonard, A., Furstenau, D., Abraham, A., Darbari, D., Tisdale, J.F. et al. Available at SSRN 4087447. (2023)
Longitudinal Study of ICET-A on Glucose Tolerance, Insulin Sensitivity and β-cell Secretion in Eleven β-Thalassemia Major Patients With Mild Iron Overload
De Sanctis V., Soliman A.T., Daar S., Tzoulis P., Di Maio S., Kattamis C. Acta Biomed. (2023)
Association of Sickle Cell Disease With Severe Maternal Morbidity
Ha, T. K., Boulet, S. L., Cotsonis, G., Geary, F., Jamieson, D. J., & Lindsay, M. Obstetrics & Gynecology, 141(1), 163-169. (2023)
Juggling Between the Cost and Value of New Therapies: Does Science Still Serve Patient Needs?
Eleftheriou, A., Angastiniotis, M., Farmakis, D., Englezos, P., Tuli, S. et al. Biomedical Journal of Scientific & Technical Research, 41(1), 3235-32351. (2023)
Overall and Complication‐free Survival in a Large Cohort of Patients With β‐Thalassemia Major Followed Over 50 Years
Forni, G.L., Gianesin, B., Musallam, K.M., Longo, F., Rosso, R., Lisi, R., et al. American Journal of Hematology. (2023)
Emergent Treatments for β-Thalassemia and Orphan Drug Legislations
Costa, E., Cappellini, M. D., Rivella, S., Chilin, et al. Drug Discovery Today, 103342. (2022)
Renal Dysfunction in Pediatric Patients in Iraq With β-Thalassemia Major and Intermedia
Mohammad G. S., Meaad K. H., Hamid J., Lamia M. Cureus. (2022)
Precision Medicine and Sickle Cell Disease
El Hoss, S., El Nemer, W., & Rees, D. C. HemaSphere, 6(9). (2022)
Does Hepcidin Tuning Have a Role Among Emerging Treatments for Thalassemia?
Longo, F., & Piga, A. Journal of Clinical Medicine, 11(17), 5119. (2022)
In Vivo Base Editing by a Single Intravenous Vector Injection for Treatment of Hemoglobinopathies
Li, C., Georgakopoulou, A., Newby, et al. JCI insight. (2022)
Luspatercept for the Treatment of Anaemia in Non-Transfusion-Dependent β-Thalassaemia (BEYOND): A Phase 2, Randomised, Double-blind, Multicentre, Placebo-controlled Trial
Taher, A. T., Cappellini, M. D., Kattamis, A., et al. The Lancet Haematology. (2022)
Luspatercept for the Treatment of Anaemia in Non-Transfusion-Dependent β-Thalassaemia (BEYOND): A Phase 2, Randomised, Double-blind, Multicentre, Placebo-controlled Trial
Taher, A. T., Cappellini, M. D., Kattamis, A., et al. The Lancet Haematology. (2022)
Nutritional Status in a Sample of Patients With β-Thalassemia Major
Lidoriki, I., Stavrou, G., Schizas, et al. Cureus14(8). (2022)
Safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Non-Transfusion Dependent α-Thalassaemia or β-Thalassaemia: An Open-label, Multicentre, Phase 2 Study
Kuo, K. H., Layton, D. M., Lal, A., et al. The Lancet, 400(10351), 493-501. (2022)
New Entity—Thalassemic Endocrine Disease: Major Beta-Thalassemia and Endocrine Involvement
Carsote, M., Vasiliu, C., Trandafir, A. I., Albu, S. E., et al. Diagnostics, 12(8), 1921. (2022)
2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia
Farmakis, D., Porter, J., Taher, A., Cappellini, M.D., Angastiniotis, M. and Eleftheriou, A. HemaSphere, 6(8). (2022).
Frequency, Pattern, and Associations of Renal Iron Accumulation in Sickle/β-Thalassemia Patients
Meloni, A., Barbuto, L., Pistoia, L., et al. Annals of Hematology, 101(9), 1941-1950. (2022)
Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
Bou-Fakhredin, R., De Franceschi, L., Motta, I., Cappellini, M. D., & Taher, A. T. Pharmaceuticals, 15(6), 753. (2022)
Promoting Adherence to Iron Chelation Treatment in Beta-Thalassemia Patients
Eziefula, C., Shah, F. T., & Anie, K. A. Patient preference and adherence, 16, p.1423. (2022).
Sickle Cell Disease Related Mortality in Brazil, 2000-2018
Santo, A. H. Transfusion and Cell Therapy, 44, 177-185. (2022)
l-glutamine, crizanlizumab, voxelotor, and cell-based therapy for adult sickle cell disease: Hype or hope?
Leibovitch, J. N., Tambe, A. V., Cimpeanu, E., Poplawska, M., Jafri, F., Dutta, D., & Lim, S. H. Blood Reviews, 100925. (2022).
Kidney Stones in Transfusion-Dependent Thalassemia: Prevalence and Risk Factors
Sayani, F. A., Lal, A., Tasian, G. E., Al Mukaddam, M., Killilea, D. W., & Fung, E. B. Open Journal of Urology, 12(4), 209-227. (2022)
Global Globin Network Consensus Paper: Classification and Stratified Roadmaps for Improved Thalassaemia Care and Prevention in 32 Countries
Halim-Fikri, B. H., Lederer, C. W., Baig, A. A., et al. .Journal of personalized medicine, 12(4), 552. (2022)
Pregnancy Outcomes Among Women Affected with Thalassemia Traits
Ruangvutilert, P., Phatihattakorn, C., Yaiyiam, C., & Panchalee, T. Archives of Gynecology and Obstetrics, 1-8. (2022)
Early Detection of Iron Overload Cardiomyopathy in Transfusion Dependent Thalassemia Patients in Sulaimaniyah City, Iraq
Ahmed, R. A., Salih, A. F., Omer, S. H., et al. Therapeutics and Clinical Risk Management, 18, 259. (2022)
Transfusion Complications in Thalassemia: Patient Knowledge and Perspectives
Patterson, S., Singleton, A., Branscomb, J., Nsonwu, V., & Spratling, R. Frontiers in medicine, 9, 772886. (2022)
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
Kanter, J., Walters, M. C., Krishnamurti, L., et al. New England Journal of Medicine, 386(7), 617-628. (2022)
Iron Overload Status in Patients with Non-Transfusion-Dependent Thalassemia in China
Huang, Y., Yang, G., Wang, M., Wei, et al. Therapeutic Advances in Hematology, 13, 20406207221084639. (2022)
Nutrition in Thalassemia: A Systematic Review of Deficiency, Relations to Morbidity, and Supplementation Recommendations
Goldberg, E. K., Lal, A., & Fung, E. B. Journal of Pediatric Hematology/Oncology, 44(1), 1-11. (2022)
Morbidity-free Survival and Hemoglobin Level in Non-Transfusion-Dependent β-Thalassemia: A 10-Year Cohort Study
Musallam, K. M., Cappellini, M. D., Daar, S., & Taher, A. T. Annals of Hematology, 101(1), 203-204. (2022)
Atrial Fibrillation in β-Thalassemia: Overview of Mechanism, Significance and Clinical Management
Guidelines for the Monitoring and Management of Iron Overload in Patients with Haemoglobinopathies and Rare Anaemias
Shah, F..T., Porter, J. B., Kaya, B. et al. British Journal of Haematology, 196, 336–350. (2022)
The Influence of Cardiovascular Risk Factors and Hypogonadism on Cardiac Outcomes in an Aging Population of Beta-Thalassemia Patients
Barbero, U., Ajassa, M., Gaglioti, C. M., Piga, A., et al. Journal of cardiovascular development and disease, 9(1), 3. (2021)
Hepatocellular Carcinoma in Transfusion Dependent Thalassemia Patients: A Review from a Clinical Perspective
Papadopoulos, N., & Koskinas, J. Hepatoma Research, 7, 77. (2021)
Evaluation of Stillbirth Among Pregnant People With Sickle Cell Trait
Canelón SP, Butts S, Boland MR. JAMA Netw Open. (2021)
Phylogenetic Analysis in the Clinical Risk Management of an Outbreak of Hepatitis C Virus Infection Among Transfused Thalassaemia Patients in Italy
Mazzucco, W., di Maio, V. C., Bronte, F., Fabeni, L., et al. Journal of Hospital Infection, 115, 51-58. (2021)
Renal Function in β-Thalassemia Major Patients Treated with Two Different Iron-Chelation Regimes
Tanous, O., Azulay, Y., Halevy, R., et al. BMC nephrology, 22(1), 1-11. (2021)
Prenatal Diagnosis Leads to Early Diagnosis of Transfusion-Dependent Thalassemia and Better Growth Outcomes
Asawasudsakorn, N., Lauhasurayotin, S., Poparn, H., et al. Global Pediatric Health, 8. (2021)
Glucose Metabolism and Insulin Response to Oral Glucose Tolerance Test (OGTT) in Prepubertal Patients with Transfusion-dependent β-Thalassemia (TDT): A Long-term Retrospective Analysis
De Sanctis, V., Soliman, A. T., Tzoulis, P., Daar, S., Di Maio, et al. Mediterranean Journal of Hematology and Infectious Diseases, 13(1). (2021)
Effect of HBB genotype on survival in a cohort of transfusion-dependent thalassemia patients in Cyprus
Kountouris, P., Michailidou, K., Christou, S., Hadjigavriel, et al. Haematologica, 106(9), 2458. (2021)
The Transfusion Management of Beta Thalassemia in the United States
Lal, A., Wong, T., Keel, S., Pagano, M., Chung, J., et al. Transfusion, 61(10), p.3027. (2021)
Pregnancy Outcomes in Women Affected by Fetal Alpha-Thalassemia: A Case Control Study
Li, J., Yan, J., Huang, Y., Wei, J., Xie, B., Zhu, M., & Jiang, W. Scientific Reports, 11(1), 1-8. (2021)
How to Facilitate Decision-Making for Hematopoietic Stem Cell Transplantation in Patients With Hemoglobinopathies. The Perspectives of Healthcare Professionals
Mekelenkamp, H., Zanten, H. V., Vries, M. D., Lankester, A., & Smiers, F. Frontiers in Pediatrics, 9, 690309. (2021)
The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease
Delgado, J., Voltz, C., Stain, M., Lapveteläinen, T., et al. HemaSphere, 5(7), e604. (2021)
Economic Evaluation of Betibeglogene Autotemcel (Beti-cel) Gene Addition Therapy in Transfusion-Dependent β-Thalassemiaon of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
Kansal, A. R., Reifsnider, O. S., Brand, S. B., Hawkins, N., et al. Journal of market access & health policy, 9(1), 1922028. (2021)
Research in Sickle Cell Disease: From Bedside to Bench to Bedside
Cisneros, G. S., & Thein, S. L. HemaSphere, 5(6). (2021)
Innovative Treatments for Rare Anemias
Cappellini, M. D., Marcon, A., Fattizzo, B., & Motta, I. HemaSphere, 5(6). (2021)
Survival and Causes of Death in 2,033 Patients with Non-Transfusion-Dependent β-Thalassemia
Musallam, K.M., Vitrano, A., Meloni, A., Pollina, S.A., Karimi, M., El-Beshlawy A. et al. Haematologica. 106(9), p.2489. (2021)
Survival and Causes of Death in 2,033 Patients with Non-Transfusion-Dependent β-Thalassemia
Musallam, K. M., Vitrano, A., Meloni, A., et al. (2021). Haematologica, 106(9), 2489. (2021)
Setting up and sustaining blood and marrow transplant services for children in middle-income economies: an experience-driven position paper on behalf of the EBMT PDWP
Faulkner, L., Verna, M., Corbacioglu, S., et al. .Bone Marrow Transplantation, 56(3), 536-543. (2021)
Hydroxyurea Use Among Children With Sickle Cell Disease at King Abdulaziz University Hospital in Jeddah City
Alzahrani, F., Albaz, G. F., AlSinan, F., et al. Cureus, 13(2). (2021)
Assessment of Subclinical Renal Glomerular and Tubular Dysfunction in Children with Beta Thalassemia Major
Mahmoud, A. A., Elian, D. M., Abd El Hady, N. M., et al. Children, 8(2), 100. (2021)
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Frangoul, H., Altshuler, D., Cappellini, M.D., Chen, Y.S., et al. New England Journal of Medicine, 384(3), pp.252-260. (2021)
Hemoglobin disorders in Europe: A Systematic Effort of Identifying and Addressing Unmet Needs and Challenges by the Thalassaemia International Federation
Angastiniotis, M., Brunetta, A. L., Constantinou, G., Knoll, E. et al. Thalassemia Reports, 11(1). (2021)
The Use of Luspatercept for Thalassemia in Adults
Cappellini, M.D. and Taher, A.T. Blood Advances, 5(1), pp.326-333. (2021)
The Prevention of Thalassemia Revisited: A Historical and Ethical Perspective by the Thalassemia International Federation
Angastiniotis, M., Petrou, M., Loukopoulos, D., Modell, B., Farmakis, D., Englezos, P. and Eleftheriou, A. Hemoglobin (2021)
Nutrition in Thalassemia: A Systematic Review of Deficiency, Relations to Morbidity, and Supplementation Recommendations
Goldberg, E. K., Lal, A., & Fung, E. B. Journal of Pediatric Hematology/Oncology, 44(1), 1-11. (2022)
Fair Pricing of Innovative Medicines: An EHA Position Paper
Hagenbeek, A., Gribben, J., Jäger, U., et al. HemaSphere, 4(5), e488. (2020)
Gene Therapy of the Hemoglobinopathies
Kunz, Joachim B.; Kulozik, Andreas E. HemaSphere. doi: 10.1097/HS9.0000000000000479 (2020)
Challenging the Erythropoiesis Paradigm in β-Thalassemia
Vinchi, F., & Vance, S. Z. HemaSphere, 4(5). (2020)
Effects of A-Tocopherol on Hemolysis and Oxidative Stress Markers on Red Blood Cells in β-Thalassemia Major
Sovira N, Lubis M, Wahidiyat PA, Suyatna FD, et al. Clin Exp Pediatr. Aug;63(8):314-320. (2020)
Recommendations for Pregnancy in Rare Inherited Anemias
Taher, A. T., Iolascon, A., Matar, C. F., Bou-Fakhredin, R., de Franceschi, L., Cappellini, M. D., et al. HemaSphere, 4(4), e446. (2020)
Update in Laboratory Diagnosis of Thalassemia
Munkongdee T, Chen P, Winichagoon P, Fucharoen S, Paiboonsukwong K. Front Mol Biosci. 10.3389/fmolb.2020.00074. PMID: 32671092; PMCID: PMC7326097. (2020)
The Changing Epidemiology of the Ageing Thalassaemia Populations: A Position Statement of the Thalassaemia International Federation
Angastiniotis, M., Eleftheriou, A., Farmakis, D. et al. European Journal of Hematology. (2020)
Sickle Cell Pain Crisis: Clinical Guidelines for the Use of Oxygen
Omoigui, S. Practical Pain Management. (2020)
Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes
Weber, L., Frati, G., Felix, T., Wollenschlaeger, C., Casini, A., Meneghini, V., et al. Science Journals. (2020)
Management of Age-associated Medical Complications in Patients with β-Thalassemia
Motta, I., Mancarella, M., Marcon, A., Vicenzi, M., & Cappellini, M. D. Expert review of hematology, 13(1), 85-94. (2020).
Challenges of Blood Transfusions in β-Thalassemia
Shah, F. T., Sayani, F., Trompeter, S., Drasar, E., & Piga, A. Blood reviews, 100588. (2019)
Understanding the Complications of Sickle Cell Disease
Tanabe P., et al. AJN The American Journal of Nursing 119.6: 26-35. (2019)
Innovative Curative Treatment of Beta-Thalassemia: Cost-efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem Cell Transplantation
Coquerelle, S., Ghardallou et al. Human gene therapy. (2019)
Reduction of Cardiac Outcomes in Thalassemia Major Thanks to a Ten-year National Italian Networking
Meloni A., Pistoia L. et al. EHA learning centre June 14. (2019)
Current Challenges in the Management of Patients with Sickle Cell Disease: A Report of the Italian Experience
Russo G., De Franceschi L., Colombatti, et al. Orphanet journal of Rare Diseases, 14(1). (2019)
Need for a Universal Thalassemia Screening Programme in India? A public health perspective
Thiyagarajan A., Bhattacharya S., Sharma N., Srivastava A., Dhar DK. J Family Med Prim Care. (2019)
The Carrier State for Sickle Cell Disease Is Not Completely Harmless
Xu, J.Z. and Thein, S.L. Haematologica, 104(6). (2019)
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