Cost–effectiveness of BTK inhibitors vs bendamustine and rituximab in chronic lymphocytic leukemia patients

Papers of special note have been highlighted as: • of interest; •• of considerable interest

References

• 1. Hillmen P, Eichhorst B, Brown JR et al. Zanubrutinib versus ibrutinib in relapsed/refractory chronic lymphocytic leukemia and small lymphocytic lymphoma: interim analysis of a randomized phase III trial. J. Clin. Oncol. 41(5), 1035–1045 (2022).
  MedlineGoogle Scholar
• 2. Yang S, Varghese AM, Sood N et al. Ethnic and geographic diversity of chronic lymphocytic leukaemia. Leukemia 35(2), 433–439 (2021).
  MedlineGoogle Scholar
• 3. Hallek M, Al-Sawaf O. Chronic lymphocytic leukemia: 2022 update on diagnostic and therapeutic procedures. Am. J. Hematol. 96(12), 1679–1705 (2021).
  MedlineGoogle Scholar
• 4. Shadman M. Diagnosis and treatment of chronic lymphocytic leukemia: a review. JAMA 329(11), 918–932 (2023). • Discusses the diagnosis and treatment process of chronic lymphocytic leukemia (CLL), and helps to understand this disease.
  Medline CASGoogle Scholar
• 5. Huber H, Edenhofer S, Von Tresckow J et al. Obinutuzumab (GA-101), ibrutinib, and venetoclax (GiVe) frontline treatment for high-risk chronic lymphocytic leukemia. Blood 139(9), 1318–1329 (2022).
  Medline CASGoogle Scholar
• 6. Xu W, Yang S, Zhou K et al. Treatment of relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma with the BTK inhibitor zanubrutinib: phase 2, single-arm, multicenter study. J. Hematol. Oncol. 13(1), 48 (2020).
  Medline CASGoogle Scholar
• 7. Ou Y, Long Y, Ji L et al. Trends in disease burden of chronic lymphocytic leukemia at the global, regional, and national levels from 1990 to 2019, and projections until 2030: a population-based epidemiologic study. Front Oncol. 12, 840616 (2022).
  MedlineGoogle Scholar
• 8. Gu D, Li J, Miao Y. Evaluating orelabrutinib as a novel treatment option for relapsed/refractory chronic lymphocytic leukemia in China. Expert Opin. Pharmacother. 23(18), 1979–1986 (2022).
  Medline CASGoogle Scholar
• 9. Wang E, Mi X, Thompson MC et al. Mechanisms of resistance to noncovalent Bruton's tyrosine kinase inhibitors. N. Engl. J. Med. 386(8), 735–743 (2022).
  Medline CASGoogle Scholar
• 10. Mendes-Bastos P, Brasileiro A, Kolkhir P et al. Bruton's tyrosine kinase inhibition-An emerging therapeutic strategy in immune-mediated dermatological conditions. Allergy 77(8), 2355–2366 (2022).
  Medline CASGoogle Scholar
• 11. Kuter DJ, Efraim M, Mayer J et al. Rilzabrutinib, an oral BTK inhibitor, in immune thrombocytopenia. N. Engl. J. Med. 386(15), 1421–1431 (2022).
  Medline CASGoogle Scholar
• 12. Mato AR, Shah NN, Jurczak W et al. Pirtobrutinib in relapsed or refractory B-cell malignancies (BRUIN): a phase 1/2 study. Lancet 397(10277), 892–901 (2021).
  Medline CASGoogle Scholar
• 13. Association HCOCM, Association HOCOCa-C, Leukemia CWGFCL. The guidelines for diagnosis and treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma in China. Zhonghua Xue Ye Xue Za Zhi 43(5), 353–358 (2022). • Summary of standard therapy with CLL patients in China.
  MedlineGoogle Scholar
• 14. Brown JR, Eichhorst B, Hillmen P et al. Zanubrutinib or ibrutinib in relapsed or refractory chronic lymphocytic leukemia. N. Engl. J. Med. 388(4), 319–332 (2023). •• Phase III trial demonstrating that zanubrutinib significantly improve progression-free survival in patients with relapsed or refractory CLL or small lymphocytic lymphoma, best supporting our article (ALPINE trials).
  Medline CASGoogle Scholar
• 15. Killock D. Zanubrutinib succeeds in head-to-head with ibrutinib in R/R CLL. Nat. Rev. Clin. Oncol. 20(2), 64 (2023).
  Google Scholar
• 16. Molica S, Tam C, Allsup D, Polliack A. Advancements in the treatment of CLL: the rise of zanubrutinib as a preferred therapeutic option. Cancers (Basel) 15(14), 3737 (2023).
  Medline CASGoogle Scholar
• 17. Rigolin GM, Olimpieri PP, Summa V et al. Outcomes in patients with chronic lymphocytic leukemia and TP53 aberration who received first-line ibrutinib: a nationwide registry study from the Italian Medicines Agency. Blood Cancer J. 13(1), 99 (2023).
  MedlineGoogle Scholar
• 18. Salmerón-Navas FJ, Barreiro-Fernández EM, Fénix-Caballero S. Adjusted indirect comparison of zanubrutinib and ibrutinib in first-line treatment of chronic lymphocytic leukemia. Farm. Hosp. doi:10.1016/j.farma.2023.07.006 (2023) (Online ahead of print).
  Google Scholar
• 19. Yang L, Qiu X, Zhao Q et al. YouTube as a platform to better understand the treatment of lymphoma using ibrutinib: a cross-sectional study. Ann. Transl. Med. 10(16), 867 (2022).
  MedlineGoogle Scholar
• 20. Martini DJ, Chen YB, Defilipp Z. Recent FDA approvals in the treatment of graft-versus-host disease. Oncologist 27(8), 685–693 (2022).
  MedlineGoogle Scholar
• 21. Sarosiek S, Sermer D, Branagan AR, Treon SP, Castillo JJ. Zanubrutinib for the treatment of adults with Waldenstrom macroglobulinemia. Expert Rev. Anticancer Ther. 22(5), 471–478 (2022).
  Medline CASGoogle Scholar
• 22. Timofeeva N, Gandhi V. ibrutinib combinations in CLL therapy: scientific rationale and clinical results. Blood Cancer J. 11(4), 79 (2021).
  MedlineGoogle Scholar
• 23. Woyach JA, Ruppert AS, Heerema NA et al. Ibrutinib regimens versus chemoimmunotherapy in older patients with untreated CLL. N. Engl. J. Med. 379(26), 2517–2528 (2018).
  Medline CASGoogle Scholar
• 24. Estupiñán HY, Berglöf A, Zain R, Smith CIE. Comparative analysis of BTK inhibitors and mechanisms underlying adverse effects. Front Cell Dev. Biol. 9, 630942 (2021).
  MedlineGoogle Scholar
• 25. Guo Y, Liu Y, Hu N et al. Discovery of zanubrutinib (BGB-3111), a novel, potent, and selective covalent inhibitor of Bruton's tyrosine kinase. J. Med. Chem. 62(17), 7923–7940 (2019).
  Medline CASGoogle Scholar
• 26. Sibaud V, Beylot-Barry M, Protin C, Vigarios E, Recher C, Ysebaert L. Dermatological toxicities of Bruton's tyrosine kinase inhibitors. Am. J. Clin. Dermatol. 21(6), 799–812 (2020).
  MedlineGoogle Scholar
• 27. Seymour JF, Kipps TJ, Eichhorst B et al. Venetoclax-rituximab in relapsed or refractory chronic lymphocytic leukemia. N. Engl. J. Med. 378(12), 1107–1120 (2018). •• The main publication for the MURANO trial containing additional information about the study design, statistical analysis and other results not reflected in the cost–effectiveness analysis.
  Medline CASGoogle Scholar
• 28. Porter DL, Hwang W-T, Frey NV et al. Chimeric antigen receptor T cells persist and induce sustained remissions in relapsed refractory chronic lymphocytic leukemia. Sci. Translat. Med. 7(303), 303ra139 (2015).
  MedlineGoogle Scholar
• 29. Su D, Wu B, Shi L. Cost–effectiveness of atezolizumab plus bevacizumab vs sorafenib as first-line treatment of unresectable hepatocellular carcinoma. JAMA Netw. Open 4(2), e210037 (2021).
  MedlineGoogle Scholar
• 30. Barbier M, Durno N, Bennison C, Ortli M, Knapp C, Schwenkglenks M. Cost–effectiveness and budget impact of venetoclax in combination with rituximab in relapsed/refractory chronic lymphocytic leukemia in Switzerland. Eur. J. Health Econ. 23(5), 837–846 (2022).
  MedlineGoogle Scholar
• 31. Tam CS, Brown JR, Kahl BS et al. Zanubrutinib versus bendamustine and rituximab in untreated chronic lymphocytic leukaemia and small lymphocytic lymphoma (SEQUOiA): a randomised, controlled, phase 3 trial. Lancet Oncol. 23(8), 1031–1043 (2022).
  Medline CASGoogle Scholar
• 32. Liu Q, Charleston MA, Richards SA, Holland BR. Performance of Akaike information criterion and Bayesian information criterion in selecting partition models and mixture models. Syst. Biol. 72(1), 92–105 (2023).
  MedlineGoogle Scholar
• 33. Portet S. A primer on model selection using the Akaike information criterion. Infect. Dis. Model 5, 111–128 (2020).
  MedlineGoogle Scholar
• 34. Selig K, Shaw P, Ankerst D. Bayesian information criterion approximations to Bayes factors for univariate and multivariate logistic regression models. Int. J. Biostat. 17(2), 241–266 (2020). • Summary of model simulation method.
  MedlineGoogle Scholar
• 35. National Bureau of Statistics. Birth rate, mortality rate and natural growth rate. https://data.stats.gov.cn/easyquery.htm?cn=C01&zb=A0302&sj=2018
  Google Scholar
• 36. Guoen L. China guidelines for pharmacoeconomic evaluations (2020). China Market Press, Bejing, China (2020). • The gold standard for pharmacoeconomic research in China.
  Google Scholar
• 37. National Bureau of Statistics. Statistical bulletin of the People's Republic of China on national economic and social development in 2021. www.stats.gov.cn/
  Google Scholar
• 38. Gong J, Su D, Shang J et al. Cost–effectiveness of tislelizumab versus docetaxel for previously treated advanced non-small-cell lung cancer in china. Front Pharmacol. 13, 830380 (2022).
  Medline CASGoogle Scholar
• 39. CDC. Report on the Nutrition and Chronic Diseases Status of Chinese Residents. People's Medical Publishing House, Beijing, China (2020).
  Google Scholar
• 40. Patel KK, isufi i, Kothari S, Davidoff AJ, Gross CP, Huntington SF. Cost–effectiveness of first-line vs third-line ibrutinib in patients with untreated chronic lymphocytic leukemia. Blood 136(17), 1946–1955 (2020). • Cost–effectiveness analysis for the USA considering a similar decision problem.
  MedlineGoogle Scholar
• 41. Wan X, Zhang Y, Tan C, Zeng X, Peng L. First-line nivolumab plus ipilimumab vs sunitinib for metastatic renal cell carcinoma: a cost–effectiveness analysis. JAMA Oncol. 5(4), 491–496 (2019).
  MedlineGoogle Scholar
• 42. Jaruratanasirikul S, Nitchot W, Wongpoowarak W, Samaeng M, Nawakitrangsan M. Population pharmacokinetics and Monte Carlo simulations of sulbactam to optimize dosage regimens in patients with ventilator-associated pneumonia caused by Acinetobacter baumannii. Eur. J. Pharm. Sci. 136, 104940 (2019).
  Medline CASGoogle Scholar
• 43. Byrd JC, Hillmen P, Ghia P et al. Acalabrutinib versus ibrutinib in previously treated chronic lymphocytic leukemia: results of the first randomized phase III trial. J. Clin. Oncol. 39(31), 3441–3452 (2021).
  Medline CASGoogle Scholar
• 44. Barr PM, Owen C, Robak T et al. Up to 8-year follow-up from RESONATE-2: first-line ibrutinib treatment for patients with chronic lymphocytic leukemia. Blood Adv. 6(11), 3440–3450 (2022).
  Medline CASGoogle Scholar
• 45. Casado LF, Hernández JÁ, Jarque i, Echave M, Casado MA, Castro A. Cost–utility analysis of idelalisib in combination with rituximab in relapsed or refractory chronic lymphocytic leukaemia. Eur. J. Haematol. 100(3), 264–272 (2018).
  Medline CASGoogle Scholar
• 46. Mandrik O, Ramos iC, Knies S, Al M, Severens JL. Cost-effectiveness of adding rituximab to fludarabine and cyclophosphamide for treatment of chronic lymphocytic leukemia in Ukraine. Cancer Manage. Res. 7, 279–289 (2015).
  MedlineGoogle Scholar
• 47. Adena M, Houltram J, Mulligan SP, Todd C, Malanos G. Modelling the cost effectiveness of rituximab in chronic lymphocytic leukaemia in first-line therapy and following relapse. Pharmaco. Economics 32(2), 193–207 (2014).
  MedlineGoogle Scholar
• 48. Vreman RA, Geenen JW, Hövels AM, Goettsch WG, Leufkens HGM, Al MJ. Phase I/II clinical trial-based early economic evaluation of acalabrutinib for relapsed chronic lymphocytic leukaemia. Appl. Health Economics Health Policy 17(6), 883–893 (2019).
  MedlineGoogle Scholar
• 49. Cai D, Shi S, Jiang S, Si L, Wu J, Jiang Y. Estimation of the cost-effective threshold of a quality-adjusted life year in China based on the value of statistical life. Eur. J. Health Econ. 23(4), 607–615 (2022). •• Contains the threshold setting for Chinese pharmacoeconomics research.
  MedlineGoogle Scholar
• 50. Zhu H, Zhu J, Zhou Y et al. Impact of the national reimbursement drug list negotiation policy on accessibility of anticancer drugs in china: an interrupted time series study. Front. Public Health 10, 921093 (2022).
  MedlineGoogle Scholar
• 51. Gale RP. Chronic lymphocytic leukemia in China. Chin. Med. J. (Engl.) 135(8), 883–886 (2022).
  Medline CASGoogle Scholar