Page 79 - 《中国药房》2025年19期

N，et al. Iron-chelating therapies in a transfusion- 市场出版社，2020：5-55.
dependent thalassaemia population in Thailand：a cost- [27] ZHANG R Q，ZHANG S，MING J，et al. A cross-
effectiveness study[J]. Clin Drug Investig，2011，31（7）： sectional study：caregiver burden and related determinants
493-505. of adult patients with β-thalassemia major in mainland
[16] KARNON J，TOLLEY K，VIEIRA J，et al. Lifetime cost- China[J]. BMC Nurs，2024，23（1）：151.
utility analyses of deferasirox in beta-thalassaemia pa‐ [28] LAWSON S E，ROBERTS I A G，AMROLIA P，et al.
tients with chronic iron overload：a UK perspective[J]. Bone marrow transplantation for beta-thalassaemia major：
Clin Drug Investig，2012，32（12）：805-815. the UK experience in two paediatric centres[J]. Br J Hae‐
[17] KESHTKARAN A，JAVANBAKHT M，SALAVATI S， matol，2003，120（2）：289-295.
et al. Cost-utility analysis of oral deferasirox versus infu‐ [29] GAZIEV J，SODANI P，POLCHI P，et al. Bone marrow
sional deferoxamine in transfusion-dependent β-thalasse‐ transplantation in adults with thalassemia：treatment and
mia patients[J]. Transfusion，2013，53（8）：1722-1729. long-term follow-up[J]. Ann N Y Acad Sci，2005，1054：
[18] BENTLEY A，GILLARD S，SPINO M，et al. Cost-utility 196-205.
analysis of deferiprone for the treatment of β-thalassaemia [30] FU B，LIAO J Y，CHEN S H，et al. CRISPR-Cas9-media-
patients with chronic iron overload：a UK perspective[J]. ted gene editing of the BCL11A enhancer for pediatric β /β 0
0
Pharmacoeconomics，2013，31（9）：807-822. transfusion-dependent β -thalassemia[J]. Nat Med，2022，
[19] HO W L，CHUNG K P，YANG S S，et al. A pharmaco- 28（8）：1573-1580.
economic evaluation of deferasirox for treating patients [31] KWIATKOWSKI J L，WALTERS M C，HONGENG S，
with iron overload caused by transfusion-dependent thala- et al. Betibeglogene autotemcel gene therapy in patients
ssemia in Taiwan[J]. J Formos Med Assoc，2013，112（4）： with transfusion-dependent，severe genotype β-thalassae‐
221-229. mia （HGB-212）：a non-randomised，multicentre，single-
[20] SRUAMSIRI R，CHAIYAKUNAPRUK N，PAKAKASAMA arm，open-label，single-dose，phase 3 trial[J]. Lancet，
S，et al. Cost-utility analysis of reduced intensity hemato‐ 2024，404（10468）：2175-2186.
poietic stem cell transplantation in adolescence and young [32] PENNELL D J，UDELSON J E，ARAI A E，et al. Cardio‐
adults with severe thalassemia compared to hypertransfu‐ vascular function and treatment in β-thalassemia major：a
sion and iron chelation program[J]. BMC Health Serv consensus statement from the American Heart Association
Res，2013，13：45. [J]. Circulation，2013，128（3）：281-308.
[21] PEPE A，ROSSI G，BENTLEY A，et al. Cost-utility analy‐ [33] ANGELUCCI E，BAROSI G，CAMASCHELLA C，et al.
sis of three iron chelators used in monotherapy for the Italian Society of Hematology practice guidelines for the
treatment of chronic iron overload in β-thalassaemia ma‐ management of iron overload in thalassemia major and re‐
jor patients：an Italian perspective[J]. Clin Drug Investig， lated disorders[J]. Haematologica，2008，93（5）：741-752.
2017，37（5）：453-464. [34] HO P J，TAY L，LINDEMAN R，et al. Australian guide‐
[22] JOHN M J，JYANI G，JINDAL A，et al. Cost effectiveness lines for the assessment of iron overload and iron chela‐
of hematopoietic stem cell transplantation compared with tion in transfusion-dependent thalassaemia major，sickle
transfusion chelation for treatment of thalassemia major cell disease and other congenital anaemias[J]. Intern Med
[J]. Biol Blood Marrow Transplant，2018，24（10）：2119- J，2011，41（7）：516-524.
2126. [35] 中华医学会血液学分会红细胞疾病贫血学组，刘容容，
[23] LI J L，WANG P，LI X，et al. Cost-utility analysis of four 李莉娟，等. 中国输血依赖型β地中海贫血诊断与治疗指
chelation regimens for β-thalassemia major：a Chinese 南：2022年版[J]. 中华血液学杂志，2022，43（11）：889-896.
perspective[J]. Mediterr J Hematol Infect Dis，2020，12 [36] 周亚丽，赖永榕，陈纯. 中国地中海贫血祛铁治疗指南：
（1）：e2020029. 2025年[J]. 中国当代儿科杂志，2025，27（4）：377-388.
[24] SAIYARSARAI P，KHORASANI E，PHOTOGERAPHY [37] DRUMMOND M，BARBIERI M，COOK J，et al. Trans‐
H，et al. Cost-utility of new film-coated tablet formulation ferability of economic evaluations across jurisdictions：
of deferasirox vs. deferoxamine among major beta- ISPOR Good Research Practices Task Force report[J].
thalassemia patients in Iran[J]. Medicine，2020，99（28）： Value Health，2009，12（4）：409-418.
e20949. [38] HEUPINK L F，PEACOCKE E F，SÆTERDAL I，et al.
[25] KANSAL A R，REIFSNIDER O S，BRAND S B，et al. Considerations for transferability of health technology as‐
Economic evaluation of betibeglogene autotemcel （Beti- sessments：a scoping review of tools，methods，and prac‐
cel） gene addition therapy in transfusion-dependent tices[J]. Int J Technol Assess Health Care，2022，38（1）：
β -thalassemia[J]. J Mark Access Health Policy，2021，9 e78.
（1）：1922028. （收稿日期：2025-04-07 修回日期：2025-08-28）
[26] 刘国恩. 中国药物经济学评价指南2020[M]. 北京：中国 （编辑：胡晓霖）

中国药房 2025年第36卷第19期 China Pharmacy 2025 Vol. 36 No. 19 · 2421 ·