Thalassemia-related complications in pediatric, adolescent, and young adult patients with transfusion-dependent thalassemia: A multicenter study in Thailand.


Journal

Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624

Informations de publication

Date de publication:
10 2023
Historique:
revised: 24 06 2023
received: 13 05 2023
accepted: 13 07 2023
medline: 24 8 2023
pubmed: 25 7 2023
entrez: 24 7 2023
Statut: ppublish

Résumé

Management of transfusion-dependent thalassemia (TDT) can be challenging due to numerous potential disease-related complications and comorbidities in particular age groups. The objective of this study was to report thalassemia-related complications and risk factors in pediatric, adolescent, and young adult patients with TDT. A multicenter web-based registry was conducted in patients with TDT aged 25 years and younger from eight university hospitals covering all parts of Thailand. Factors significantly associated with each complication were analyzed by logistic regression methods. Of 605 patients, 267 thalassemia-related complications were reported from 231 pediatric, adolescent, and young adult patients with TDT patients (38.2%). The most common complications were infections, followed by cholelithiasis and growth failure. Splenectomy and elevated pre-transfusion hemoglobin were statistically significant risk factors for infections (adjusted odds ratio [AOR] = 2.3, 95% confidence interval [CI]: 1.2-4.5, p-value = .01 and AOR = 1.5, 95% CI: 1.2-1.7, p-value < .005, respectively). There were two statistically significant risk factors conferred endocrinopathies, including older age (AOR = 1.06, 95% CI: 1.01-1.1, p-value = .01) and being male (AOR = 2.4, 95% CI: 1.4-4.0, p-value = .002). Nearly 40% of the patients in this cohort had thalassemia-related complications. Periodic surveillance and optimal care for respective complications may minimize comorbidities in pediatric, adolescent, and young adult patients with TDT.

Identifiants

pubmed: 37488065
doi: 10.1002/pbc.30599
doi:

Types de publication

Multicenter Study Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

e30599

Subventions

Organisme : NCATS NIH HHS
ID : UL1 TR002535
Pays : United States

Informations de copyright

© 2023 Wiley Periodicals LLC.

Références

Weatherall DJ. The inherited diseases of hemoglobin are an emerging global health burden. Blood. 2010;115:4331-4336.
Weatherall DJ. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. Nat Rev Genet. 2001;2:245-255.
De Sanctis V, Eleftheriou A, Malaventura C. Prevalence of endocrine complications and short stature in patients with thalassaemia major: a multicenter study by the Thalassaemia International Federation (TIF). Pediatr Endocrinol Rev. 2004;2(2):249-255.
De Sanctis V, Soliman AT, Elsedfy H, et al. Growth and endocrine disorders in thalassemia: the international network on endocrine complications in thalassemia (I-CET) position statement and guidelines. Indian J Endocrinol Metab. 2013;17:8-18.
Shah F, Huey K, Deshpande S, et al. Relationship between serum ferritin and outcomes in β-thalassemia: a systematic literature review. J Clin Med. 2022;11.
Chuncharunee S, Teawtrakul N, Siritanaratkul N, Chueamuangphan N. Review of disease-related complications and management in adult patients with thalassemia: a multi-center study in Thailand. PLoS One. 2019;14:e0214148.
Teawtrakul N, Jetsrisuparb A, Pongudom S, et al. Epidemiologic study of major complications in adolescent and adult patients with thalassemia in Northeastern Thailand: the E-SAAN study phase I. Hematology. 2018;23:55-60.
Tantiworawit A, Tapanya S, Phrommintikul A, et al. Prevalence and risk factors for cardiac iron overload and cardiovascular complications among patients with thalassemia in Northern Thailand. Southeast Asian J Trop Med Public Health. 2016;47:1335-1342.
Hassan T, Zakaria M, Fathy M, et al. Association between genotype and disease complications in Egyptian patients with beta thalassemia: a cross-sectional study. Sci Rep. 2018;8:17730.
Saliba AN, El Rassi F, Taher AT. Clinical monitoring and management of complications related to chelation therapy in patients with β-thalassemia. Expert Rev Hematol. 2016;9:151-168.
Thuret I, Pondarré C, Loundou A, et al. Complications and treatment of patients with β-thalassemia in France: results of the National Registry. Haematologica. 2010;95:724-729.
Cunningham MJ, Macklin EA, Neufeld EJ, Cohen AR, Thalassemia Clinical Research Network. Complications of beta-thalassemia major in North America. Blood. 2004;104:34-39.
Koohi F, Kazemi T, Miri-Moghaddam E. Cardiac complications and iron overload in beta thalassemia major patients-a systematic review and meta-analysis. Ann Hematol. 2019;98:1323-1331.
Carsote M, Vasiliu C, Trandafir AI, et al. New entity-thalassemic endocrine disease: major beta-thalassemia and endocrine involvement. Diagnostics (Basel). 2022;12(8):1921.
Arab-Zozani M, Kheyrandish S, Rastgar A, Miri-Moghaddam E. A systematic review and meta-analysis of stature growth complications in β-thalassemia major patients. Ann Glob Health. 2021;87:48.
Casale M, Forni GL, Cassinerio E, et al. Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multi-center nation-wide cohort. Haematologica. 2022;107:467-477.
Taher AT, Cappellini MD, Musallam KM. Development of a thalassemia-related thrombosis risk scoring system. Am J Hematol. 2019;94:E207-E209.
Farmakis D, Porter J, Taher A, Domenica CM, Angastiniotis M, Eleftheriou A. 2021 Thalassaemia International Federation guidelines for the management of transfusion-dependent thalassemia. Hemasphere. 2022;6:e732.
Harris PA, Taylor R, Thielke R, Payne J, Gonzalez N, Conde JG. Research electronic data capture (REDCap-a metadata-driven methodology and workflow process for providing translational research informatics support. J Biomed Inform. 2009;42:377-381.
Ommen SR, Nishimura RA, Hurrell DG, Klarich KW. Assessment of right atrial pressure with 2-dimensional and Doppler echocardiography: a simultaneous catheterization and echocardiographic study. Mayo Clin Proc. 2000;75:24-29.
Teawtrakul N, Pussadhamma B, Ungprasert P, et al. A risk score for predicting pulmonary hypertension in patients with non-transfusion-dependent thalassemia in northeastern Thailand: the E-SAAN score. Hematology. 2015;20:416-421.
Ho KK, Pinsky JL, Kannel WB, Levy D. The epidemiology of heart failure: the Framingham Study. J Am Coll Cardiol. 1993;22:A6-A13.
Kanis JA, Melton LJ, Christiansen C, Johnston CC, Khaltaev N. The diagnosis of osteoporosis. J Bone Miner Res. 1994;9:1137-1141.
American Diabetes Association. Diagnosis and classification of diabetes mellitus. Diabetes Care. 2014;37(1:S81-S90.
Yaghobi M, Miri-Moghaddam E, Majid N, Bazi A, Navidian A, Kalkali A. Complications of transfusion-dependent β-thalassemia patients in Sistan and Baluchistan, South-East of Iran. Int J Hematol Oncol Stem Cell Res. 2017;11:268-272.
Dixit N, Shaw CK, Varshney GA, Kumar R, Saini PA, Verma P. Endocrinal complications in children and adolescents with thalassemia major in Central India: an observational study. Indian J Pediatr. 2022;89:983-988.
Mahmoud RA, Khodeary A, Farhan MS. Detection of endocrine disorders in young children with multi-transfused thalassemia major. Ital J Pediatr. 2021;47:165.
Upadya SH, Rukmini MS, Sundararajan S, Baliga BS, Kamath N. Thyroid function in chronically transfused children with beta thalassemia major: a cross-sectional hospital based study. Int J Pediatr. 2018;2018:9071213.
Lee KT, Lim SL, Goh AS. Prevalence of endocrine complications in transfusion dependent thalassemia in Hospital Pulau Pinang: a pilot study. Med J Malaysia. 2020;75:33-37.
Mattia L, Samperi I, Monti S, Toscano V, Pugliese G, Poggi M. The quality of life of thalassemic patients: the role of endocrine defect compensation. Endocr Metab Immune Disord Drug Targets. 2021;21(12):2147-2158.
Etemad K, Mohseni P, Aghighi M, et al. Quality of life and related factors in β-thalassemia patients. Hemoglobin. 2021;45:245-249.
Skordis N, Kyriakou A. The multifactorial origin of growth failure in thalassaemia. Pediatr Endocrinol Rev. 2011;8((2):271-277.
Kyriakou A, Skordis N. Thalassaemia and aberrations of growth and puberty. Mediterr J Hematol Infect Dis. 2009;1:e2009003.
Marsella M, Pepe A, Borgna-Pignatti C. Better survival and less cardiac morbidity in female patients with thalassemia major: a review of the literature. Ann N Y Acad Sci. 2010;1202:129-133.
Nokeaingtong K, Charoenkwan P, Silvilairat S, Saekho S, Pongprot Y, Dejkhamron P. A longitudinal study of growth and relation with anemia and iron overload in pediatric patients with transfusion-dependent thalassemia. J Pediatr Hematol Oncol. 2016;38:457-462.

Auteurs

Pacharapan Surapolchai (P)

Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine, Thammasat University, Pathum Thani, Thailand.

Duantida Songdej (D)

Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.

Chattree Hantaweepant (C)

Division of Hematology, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.

Adisak Tantiworawit (A)

Department of Internal Medicine, Division of Hematology, Chiang Mai University, Chiang Mai, Thailand.
Thalassemia and Hematology Center, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand.

Pimlak Charoenkwan (P)

Thalassemia and Hematology Center, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand.
Department of Pediatrics, Division of Hematology and Oncology, Chiang Mai University, Chiang Mai, Thailand.

Supanun Lauhasurayotin (S)

Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.

Kitti Torcharus (K)

Division of Pediatric Hematology/Oncology, Department of Pediatrics, Phramongkutklao College of Medicine, Bangkok, Thailand.

Pornpun Sripornsawan (P)

Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine, Prince of Songkla University, Songkla, Thailand.

Pranee Sutcharitchan (P)

Division of Hematology, Department of Internal Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand.

Patcharee Konwilaisak (P)

Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand.

Supawee Saengboon (S)

Division of Hematology, Department of Internal Medicine, Faculty of Medicine, Thammasat University, Pathum Thani, Thailand.

Bunchoo Pongtanakul (B)

Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.

Nattiya Teawtrakul (N)

Division of Hematology, Department of Internal Medicine, Srinagarind Hospital, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand.

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