Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East

被引:8
作者
El-Beshlawy, Amal [1 ]
Dewedar, Hany [2 ]
Hindawi, Salwa [3 ]
Alkindi, Salam [4 ]
Tantawy, Azza A. [5 ]
Yassin, Mohamed A. [6 ]
Taher, Ali T. [7 ,8 ]
机构
[1] Cairo Univ, Fac Med, Dept Pediat Hematol, Cairo, Egypt
[2] Latifa Hosp, Thalassemia Ctr, Dubai, U Arab Emirates
[3] King Abdulaziz Univ, Fac Med, Dept Hematol, Jeddah, Saudi Arabia
[4] Sultan Qaboos Univ Hosp, Dept Hematol, Muscat, Oman
[5] Ain Shams Univ, Fac Med, Dept Pediat, Cairo, Egypt
[6] Hamad Med Corp, Natl Ctr Canc Care & Res, Dept Hematol & Oncol, Doha, Qatar
[7] Amer Univ Beirut, Med Ctr, Dept Internal Med, Beirut, Lebanon
[8] Amer Univ Beirut, Naef K Basile Canc Inst, Med Ctr, Hematol & Oncol,Dept Internal Med,Res, POB 11-0236, Beirut 11072020, Lebanon
关键词
Thalassemia; Complications; Transfusion; iron chelation; Management; STEM-CELL TRANSPLANTATION; GENETIC-COUNSELING PROGRAM; MAJOR PATIENTS RELATION; PEDIATRIC-PATIENTS; IRON CHELATION; PULMONARY-HYPERTENSION; FOLLOW-UP; DEFERASIROX; COMPLICATIONS; PREVALENCE;
D O I
10.1016/j.blre.2023.101138
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
beta-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators and more novel ineffective erythropoiesistargeted therapy. In this review, we provide a practical overview of management for patients with transfusiondependent beta-thalassemia, primarily driven by such experiences, with the aim of transferring knowledge to colleagues in other regions facing similar challenges.
引用
收藏
页数:10
相关论文
共 130 条
  • [11] Prevalence and distribution of iron overload in patients with transfusion-dependent anemias differs across geographic regions: results from the CORDELIA study
    Aydinok, Yesim
    Porter, John B.
    Piga, Antonio
    Elalfy, Mohsen
    El-Beshlawy, Amal
    Kilinc, Yurdanur
    Viprakasit, Vip
    Yesilipek, Akif
    Habr, Dany
    Quebe-Fehling, Erhard
    Pennell, Dudley J.
    [J]. EUROPEAN JOURNAL OF HAEMATOLOGY, 2015, 95 (03) : 244 - 253
  • [12] Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload
    Aydinok, Yesim
    Kattamis, Antonis
    Cappellini, M. Domenica
    El-Beshlawy, Amal
    Origa, Raffaella
    Elalfy, Mohsen
    Kilinc, Yurdanur
    Perrotta, Silverio
    Karakas, Zeynep
    Viprakasit, Vip
    Habr, Dany
    Constantinovici, Niculae
    Shen, Junwu
    Porter, John B.
    [J]. BLOOD, 2015, 125 (25) : 3868 - 3877
  • [13] Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010
    Baronciani, D.
    Angelucci, E.
    Potschger, U.
    Gaziev, J.
    Yesilipek, A.
    Zecca, M.
    Orofino, M. G.
    Giardini, C.
    Al-Ahmari, A.
    Marktel, S.
    de la Fuente, J.
    Ghavamzadeh, A.
    Hussein, A. A.
    Targhetta, C.
    Pilo, F.
    Locatelli, F.
    Dini, G.
    Bader, P.
    Peters, C.
    [J]. BONE MARROW TRANSPLANTATION, 2016, 51 (04) : 536 - 541
  • [14] Syrian Refugees and Their Impact on Health Service Delivery in the Pediatric Hematology/Oncology Clinics Across Canada
    Barry, Rachid M.
    Chretien, Christine
    Kirby, Melanie
    Gallant, Gloria
    Leppington, Sarah
    Robitaille, Nancy
    Corriveau-Bourque, Catherine
    Stoffman, Jayson
    Wu, John
    Leaker, Michael
    Klaassen, Robert J.
    [J]. JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY, 2020, 42 (02) : E107 - E109
  • [15] Hematopoietic stem cell transplantation in Lebanon: first comprehensive report
    Bazarbachi, A.
    Hatoum, H. A.
    Mugharbel, A.
    Otrock, Z. K.
    Yassine, N.
    Muwakkit, S.
    Salem, Z.
    Shebbo, W.
    Jisr, T.
    Abboud, M.
    Ibrahim, A.
    [J]. BONE MARROW TRANSPLANTATION, 2008, 42 (Suppl 1) : S96 - S102
  • [16] Serum ferritin levels and endocrinopathy in medically treated patients with β thalassemia major
    Belhoul, Khawla M.
    Bakir, Maisam L.
    Saned, Mohamed-SalahEldin
    Kadhim, Ahmed M. A.
    Musallam, Khaled M.
    Taher, Ali T.
    [J]. ANNALS OF HEMATOLOGY, 2012, 91 (07) : 1107 - 1114
  • [17] Premarital screening and genetic counseling program: Studies from an endogamous population
    Bener, Abdulbari
    Al-Mulla, Mariam
    Clarke, Angus
    [J]. INTERNATIONAL JOURNAL OF APPLIED AND BASIC MEDICAL RESEARCH, 2019, 9 (01) : 20 - 26
  • [18] Systematic Literature Review of the Burden of Disease and Treatment for Transfusion-dependent β-Thalassemia
    Betts, Marissa
    Flight, Patrick A.
    Paramore, L. Clark
    Tian, Li
    Milenkovic, Dusan
    Sheth, Sujit
    [J]. CLINICAL THERAPEUTICS, 2020, 42 (02) : 322 - +
  • [19] Survival and complications in thalassemia
    Borgna-Pignatti, C
    Cappellini, MD
    De Stefano, P
    Del Vecchio, GC
    Forni, GL
    Gamberini, MR
    Ghilardi, R
    Origa, R
    Piga, A
    Romeo, MA
    Zhao, H
    Cnaan, A
    [J]. COOLEY'S ANEMIA EIGHTH SYMPOSIUM, 2005, 1054 : 40 - 47
  • [20] A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
    Cappellini, M. D.
    Viprakasit, V.
    Taher, A. T.
    Georgiev, P.
    Kuo, K. H. M.
    Coates, T.
    Voskaridou, E.
    Liew, H. -K.
    Pazgal-Kobrowski, I.
    Forni, G. L.
    Perrotta, S.
    Khelif, A.
    Lal, A.
    Kattamis, A.
    Vlachaki, E.
    Origa, R.
    Aydinok, Y.
    Bejaoui, M.
    Ho, P. J.
    Chew, L. -P.
    Bee, P. -C.
    Lim, S. -M.
    Lu, M. -Y.
    Tantiworawit, A.
    Ganeva, P.
    Gercheva, L.
    Shah, F.
    Neufeld, E. J.
    Thompson, A.
    Laadem, A.
    Shetty, J. K.
    Zou, J.
    Zhang, J.
    Miteva, D.
    Zinger, T.
    Linde, P. G.
    Sherman, M. L.
    Hermine, O.
    Porter, J.
    Piga, A.
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2020, 382 (13) : 1219 - 1231