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Transfusion Dependent Thalassemia

MedGen UID:
1755933
•Concept ID:
C4760934
•
Disease or Syndrome
Synonym: Transfusion-dependent Thalassemia

Definition

Thalassemia that results in severe anemia and requires regular blood transfusions for patient survival. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVTransfusion Dependent Thalassemia

Professional guidelines

PubMed

Thalassemia: Common Clinical Queries in Management.
Lal A, Bansal D
Indian J Pediatr 2020 Jan;87(1):75-81. Epub 2019 Oct 16 doi: 10.1007/s12098-019-03065-5. PMID: 31620986
Clinical Classification, Screening and Diagnosis for Thalassemia.
Viprakasit V, Ekwattanakit S
Hematol Oncol Clin North Am 2018 Apr;32(2):193-211. doi: 10.1016/j.hoc.2017.11.006. PMID: 29458726
Non-Transfusion-Dependent Thalassemia: An Update on Complications and Management.
Sleiman J, Tarhini A, Bou-Fakhredin R, Saliba AN, Cappellini MD, Taher AT
Int J Mol Sci 2018 Jan 8;19(1) doi: 10.3390/ijms19010182. PMID: 29316681Free PMC Article

Recent clinical studies

Etiology

Anemia: Microcytic Anemia.
Rampon K
FP Essent 2023 Jul;530:12-16. PMID: 37390396
Non-Transfusion-Dependent Thalassemia: A Panoramic Review.
Shash H
Medicina (Kaunas) 2022 Oct 21;58(10) doi: 10.3390/medicina58101496. PMID: 36295656Free PMC Article
Challenges of blood transfusions in β-thalassemia.
Shah FT, Sayani F, Trompeter S, Drasar E, Piga A
Blood Rev 2019 Sep;37:100588. Epub 2019 Jul 6 doi: 10.1016/j.blre.2019.100588. PMID: 31324412
Morbidities in non-transfusion-dependent thalassemia.
Saliba AN, Taher AT
Ann N Y Acad Sci 2016 Mar;1368(1):82-94. doi: 10.1111/nyas.13083. PMID: 27186941
Hypercoagulability in non-transfusion-dependent thalassemia.
Cappellini MD, Musallam KM, Poggiali E, Taher AT
Blood Rev 2012 Apr;26 Suppl 1:S20-3. doi: 10.1016/S0268-960X(12)70007-3. PMID: 22631037

Diagnosis

Diagnosis and treatment of cardiac iron overload in transfusion-dependent thalassemia patients.
Siri-Angkul N, Chattipakorn SC, Chattipakorn N
Expert Rev Hematol 2018 Jun;11(6):471-479. Epub 2018 May 18 doi: 10.1080/17474086.2018.1476134. PMID: 29754517
Fertility and Pregnancy in Women with Transfusion-Dependent Thalassemia.
Carlberg KT, Singer ST, Vichinsky EP
Hematol Oncol Clin North Am 2018 Apr;32(2):297-315. doi: 10.1016/j.hoc.2017.11.004. PMID: 29458733
Clinical Classification, Screening and Diagnosis for Thalassemia.
Viprakasit V, Ekwattanakit S
Hematol Oncol Clin North Am 2018 Apr;32(2):193-211. doi: 10.1016/j.hoc.2017.11.006. PMID: 29458726
β-Thalassemia intermedia: a comprehensive overview and novel approaches.
Asadov C, Alimirzoeva Z, Mammadova T, Aliyeva G, Gafarova S, Mammadov J
Int J Hematol 2018 Jul;108(1):5-21. Epub 2018 Jan 29 doi: 10.1007/s12185-018-2411-9. PMID: 29380178
Non-transfusion-dependent thalassemias.
Musallam KM, Rivella S, Vichinsky E, Rachmilewitz EA
Haematologica 2013 Jun;98(6):833-44. doi: 10.3324/haematol.2012.066845. PMID: 23729725Free PMC Article

Therapy

Combination chelation therapy.
Aydinok Y
Ann N Y Acad Sci 2023 Nov;1529(1):33-41. Epub 2023 Aug 18 doi: 10.1111/nyas.15052. PMID: 37594980
Hematopoietic Stem Cell Transplantation in Thalassemia.
Algeri M, Lodi M, Locatelli F
Hematol Oncol Clin North Am 2023 Apr;37(2):413-432. doi: 10.1016/j.hoc.2022.12.009. PMID: 36907612
Non-Transfusion-Dependent Thalassemia: An Update on Complications and Management.
Sleiman J, Tarhini A, Bou-Fakhredin R, Saliba AN, Cappellini MD, Taher AT
Int J Mol Sci 2018 Jan 8;19(1) doi: 10.3390/ijms19010182. PMID: 29316681Free PMC Article
Morbidities in non-transfusion-dependent thalassemia.
Saliba AN, Taher AT
Ann N Y Acad Sci 2016 Mar;1368(1):82-94. doi: 10.1111/nyas.13083. PMID: 27186941
Deferiprone.
Piga A, Roggero S, Salussolia I, Massano D, Serra M, Longo F
Ann N Y Acad Sci 2010 Aug;1202:75-8. doi: 10.1111/j.1749-6632.2010.05586.x. PMID: 20712776

Prognosis

The association between pre-transfusion hemoglobin levels and thalassemia complications.
Wanchaitanawong W, Tantiworawit A, Piriyakhuntorn P, Rattanathammethee T, Hantrakool S, Chai-Adisaksopha C, Rattarittamrong E, Norasetthada L, Niprapan P, Fanhchaksai K, Charoenkwan P
Hematology 2021 Dec;26(1):1-8. doi: 10.1080/16078454.2020.1856513. PMID: 33357151
Thalassemia: Common Clinical Queries in Management.
Lal A, Bansal D
Indian J Pediatr 2020 Jan;87(1):75-81. Epub 2019 Oct 16 doi: 10.1007/s12098-019-03065-5. PMID: 31620986
Adrenal insufficiency in non-transfusion-dependent α-thalassemia.
Joradol N, Supornsilchai V, Tongmeesee S, Tanyong B, Aphikulchatkit Y, Sosothikul D
Pediatr Int 2017 Nov;59(11):1135-1139. Epub 2017 Sep 12 doi: 10.1111/ped.13387. PMID: 28779493
The definition and epidemiology of non-transfusion-dependent thalassemia.
Weatherall DJ
Blood Rev 2012 Apr;26 Suppl 1:S3-6. doi: 10.1016/S0268-960X(12)70003-6. PMID: 22631040
Hemoglobin e syndromes.
Vichinsky E
Hematology Am Soc Hematol Educ Program 2007:79-83. doi: 10.1182/asheducation-2007.1.79. PMID: 18024613

Clinical prediction guides

Thalassemia: Common Clinical Queries in Management.
Lal A, Bansal D
Indian J Pediatr 2020 Jan;87(1):75-81. Epub 2019 Oct 16 doi: 10.1007/s12098-019-03065-5. PMID: 31620986
Iron Chelation Therapy as a Modality of Management.
Aydinok Y
Hematol Oncol Clin North Am 2018 Apr;32(2):261-275. Epub 2018 Jan 17 doi: 10.1016/j.hoc.2017.12.002. PMID: 29458731
Adrenal insufficiency in non-transfusion-dependent α-thalassemia.
Joradol N, Supornsilchai V, Tongmeesee S, Tanyong B, Aphikulchatkit Y, Sosothikul D
Pediatr Int 2017 Nov;59(11):1135-1139. Epub 2017 Sep 12 doi: 10.1111/ped.13387. PMID: 28779493
The importance of nutrition for health in patients with transfusion-dependent thalassemia.
Fung EB
Ann N Y Acad Sci 2016 Mar;1368(1):40-8. Epub 2016 Jan 29 doi: 10.1111/nyas.13003. PMID: 26824448
Clinical pharmacology of deferasirox.
Tanaka C
Clin Pharmacokinet 2014 Aug;53(8):679-94. doi: 10.1007/s40262-014-0151-4. PMID: 24996374

Recent systematic reviews

Adherence to Iron Chelation Therapy among Adults with Thalassemia: A Systematic Review.
Locke M, Reddy PS, Badawy SM
Hemoglobin 2022 Jul;46(4):201-213. Epub 2022 Aug 5 doi: 10.1080/03630269.2022.2072320. PMID: 35930250Free PMC Article
Prevalence of transfusion-transmitted infections in multiple blood transfusion-dependent thalassemic patients in Asia: A systemic review.
Riaz M, Abbas M, Rasool G, Baig IS, Mahmood Z, Munir N, Mahmood Tahir I, Ali Shah SM, Akram M
Int J Immunopathol Pharmacol 2022 Jan-Dec;36:3946320221096909. doi: 10.1177/03946320221096909. PMID: 35452334Free PMC Article
Fracture prevalence in thalassemia: a systematic review and meta-analysis.
Charoenngam N, Rittiphairoj T, Ponvilawan B
Arch Osteoporos 2021 Nov 13;16(1):171. doi: 10.1007/s11657-021-01026-0. PMID: 34773506
Beta-thalassemia: renal complications and mechanisms: a narrative review.
Demosthenous C, Vlachaki E, Apostolou C, Eleftheriou P, Kotsiafti A, Vetsiou E, Mandala E, Perifanis V, Sarafidis P
Hematology 2019 Dec;24(1):426-438. doi: 10.1080/16078454.2019.1599096. PMID: 30947625
Cardiac complications and iron overload in beta thalassemia major patients-a systematic review and meta-analysis.
Koohi F, Kazemi T, Miri-Moghaddam E
Ann Hematol 2019 Jun;98(6):1323-1331. Epub 2019 Feb 7 doi: 10.1007/s00277-019-03618-w. PMID: 30729283

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