Cost-effectiveness of adding rituximab to splenectomy and romiplostim for treating steroid-resistant idiopathic thrombocytopenic purpura in adults

Kayoko Kikuchi, Yoshitaka Miyakawa, Shunya Ikeda, Yuji Sato, Toru Takebayashi

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

Background: Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease in which the platelet count falls to <100 × 109 /L. Corticosteroids are recommended as the first-line treatment, splenectomy is recommended as the second-line treatment, and thrombopoietin receptor agonists (TPO-RAs) and rituximab are recommended as the third-line treatments for ITP in Japanese ITP treatment guidelines. However, in Japan, rituximab is not eligible for reimbursement for the treatment of ITP. The cost-effectiveness of ITP treatment has not been investigated in Japan. Therefore, in this study, the cost-effectiveness of adding rituximab treatment to the existing treatments indicated for ITP in Japan, namely splenectomy and the TPO-RA romiplostim, was investigated based on the scenario that rituximab is eligible for reimbursement in Japan as a treatment for ITP. Methods: The efficacy endpoint was set as the number of years with a platelet count ?30 × 109/L. The analysis was conducted from the healthcare payer's perspective. If the first treatment is ineffective or relapse occurs, then the patient is given the following treatment. The analyzed treatment order consisted of three patterns: Splenectomy-romiplostim (sequence 1), splenectomy-romiplostim-rituximab (sequence 2), and splenectomy-rituximab-romiplostim (sequence 3). A Markov model was built for ITP, and the analysis period was set as 2 years. The discount rate was an annual rate of 2%. Sensitivity analyses of the efficacy of splenectomy, romiplostim, and rituximab; treatment cost; and romiplostim dose were performed. Results: The expected costs per patient over a 2-year period for sequences 1, 2, and 3 were USD 40,980, USD 39,822, and USD 33,551, respectively. The expected years with a platelet count ≤30 × 109/L for the three sequences were 1.75, 1.79, and 1.78 years, respectively. The sensitivity analyses illustrated that the results of the base case analysis were robust. Conclusions: Adding rituximab to standard treatment for ITP (sequences 2-3) is less costly and marginally more effective than standard therapy in adults. According to the study results, if rituximab is reimbursed for the treatment of ITP in Japan, medical expenses are expected to decline.

Original languageEnglish
Article number2
JournalBMC Health Services Research
Volume15
Issue number1
DOIs
Publication statusPublished - 2015 Jan 22

Fingerprint

Idiopathic Thrombocytopenic Purpura
Splenectomy
Cost-Benefit Analysis
Steroids
Japan
Therapeutics
Thrombopoietin Receptors
Platelet Count
Rituximab
romiplostim
Health Care Costs
Autoimmune Diseases

Keywords

  • Cost-effectiveness
  • ITP
  • Rituximab
  • Romiplostim
  • Splenectomy

ASJC Scopus subject areas

  • Health Policy

Cite this

Cost-effectiveness of adding rituximab to splenectomy and romiplostim for treating steroid-resistant idiopathic thrombocytopenic purpura in adults. / Kikuchi, Kayoko; Miyakawa, Yoshitaka; Ikeda, Shunya; Sato, Yuji; Takebayashi, Toru.

In: BMC Health Services Research, Vol. 15, No. 1, 2, 22.01.2015.

Research output: Contribution to journalArticle

@article{171959d96f2c4c16b7a11e1620fb9f1d,
title = "Cost-effectiveness of adding rituximab to splenectomy and romiplostim for treating steroid-resistant idiopathic thrombocytopenic purpura in adults",
abstract = "Background: Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease in which the platelet count falls to <100 × 109 /L. Corticosteroids are recommended as the first-line treatment, splenectomy is recommended as the second-line treatment, and thrombopoietin receptor agonists (TPO-RAs) and rituximab are recommended as the third-line treatments for ITP in Japanese ITP treatment guidelines. However, in Japan, rituximab is not eligible for reimbursement for the treatment of ITP. The cost-effectiveness of ITP treatment has not been investigated in Japan. Therefore, in this study, the cost-effectiveness of adding rituximab treatment to the existing treatments indicated for ITP in Japan, namely splenectomy and the TPO-RA romiplostim, was investigated based on the scenario that rituximab is eligible for reimbursement in Japan as a treatment for ITP. Methods: The efficacy endpoint was set as the number of years with a platelet count ?30 × 109/L. The analysis was conducted from the healthcare payer's perspective. If the first treatment is ineffective or relapse occurs, then the patient is given the following treatment. The analyzed treatment order consisted of three patterns: Splenectomy-romiplostim (sequence 1), splenectomy-romiplostim-rituximab (sequence 2), and splenectomy-rituximab-romiplostim (sequence 3). A Markov model was built for ITP, and the analysis period was set as 2 years. The discount rate was an annual rate of 2{\%}. Sensitivity analyses of the efficacy of splenectomy, romiplostim, and rituximab; treatment cost; and romiplostim dose were performed. Results: The expected costs per patient over a 2-year period for sequences 1, 2, and 3 were USD 40,980, USD 39,822, and USD 33,551, respectively. The expected years with a platelet count ≤30 × 109/L for the three sequences were 1.75, 1.79, and 1.78 years, respectively. The sensitivity analyses illustrated that the results of the base case analysis were robust. Conclusions: Adding rituximab to standard treatment for ITP (sequences 2-3) is less costly and marginally more effective than standard therapy in adults. According to the study results, if rituximab is reimbursed for the treatment of ITP in Japan, medical expenses are expected to decline.",
keywords = "Cost-effectiveness, ITP, Rituximab, Romiplostim, Splenectomy",
author = "Kayoko Kikuchi and Yoshitaka Miyakawa and Shunya Ikeda and Yuji Sato and Toru Takebayashi",
year = "2015",
month = "1",
day = "22",
doi = "10.1186/s12913-015-0681-y",
language = "English",
volume = "15",
journal = "BMC Health Services Research",
issn = "1472-6963",
publisher = "BioMed Central",
number = "1",

}

TY - JOUR

T1 - Cost-effectiveness of adding rituximab to splenectomy and romiplostim for treating steroid-resistant idiopathic thrombocytopenic purpura in adults

AU - Kikuchi, Kayoko

AU - Miyakawa, Yoshitaka

AU - Ikeda, Shunya

AU - Sato, Yuji

AU - Takebayashi, Toru

PY - 2015/1/22

Y1 - 2015/1/22

N2 - Background: Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease in which the platelet count falls to <100 × 109 /L. Corticosteroids are recommended as the first-line treatment, splenectomy is recommended as the second-line treatment, and thrombopoietin receptor agonists (TPO-RAs) and rituximab are recommended as the third-line treatments for ITP in Japanese ITP treatment guidelines. However, in Japan, rituximab is not eligible for reimbursement for the treatment of ITP. The cost-effectiveness of ITP treatment has not been investigated in Japan. Therefore, in this study, the cost-effectiveness of adding rituximab treatment to the existing treatments indicated for ITP in Japan, namely splenectomy and the TPO-RA romiplostim, was investigated based on the scenario that rituximab is eligible for reimbursement in Japan as a treatment for ITP. Methods: The efficacy endpoint was set as the number of years with a platelet count ?30 × 109/L. The analysis was conducted from the healthcare payer's perspective. If the first treatment is ineffective or relapse occurs, then the patient is given the following treatment. The analyzed treatment order consisted of three patterns: Splenectomy-romiplostim (sequence 1), splenectomy-romiplostim-rituximab (sequence 2), and splenectomy-rituximab-romiplostim (sequence 3). A Markov model was built for ITP, and the analysis period was set as 2 years. The discount rate was an annual rate of 2%. Sensitivity analyses of the efficacy of splenectomy, romiplostim, and rituximab; treatment cost; and romiplostim dose were performed. Results: The expected costs per patient over a 2-year period for sequences 1, 2, and 3 were USD 40,980, USD 39,822, and USD 33,551, respectively. The expected years with a platelet count ≤30 × 109/L for the three sequences were 1.75, 1.79, and 1.78 years, respectively. The sensitivity analyses illustrated that the results of the base case analysis were robust. Conclusions: Adding rituximab to standard treatment for ITP (sequences 2-3) is less costly and marginally more effective than standard therapy in adults. According to the study results, if rituximab is reimbursed for the treatment of ITP in Japan, medical expenses are expected to decline.

AB - Background: Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disease in which the platelet count falls to <100 × 109 /L. Corticosteroids are recommended as the first-line treatment, splenectomy is recommended as the second-line treatment, and thrombopoietin receptor agonists (TPO-RAs) and rituximab are recommended as the third-line treatments for ITP in Japanese ITP treatment guidelines. However, in Japan, rituximab is not eligible for reimbursement for the treatment of ITP. The cost-effectiveness of ITP treatment has not been investigated in Japan. Therefore, in this study, the cost-effectiveness of adding rituximab treatment to the existing treatments indicated for ITP in Japan, namely splenectomy and the TPO-RA romiplostim, was investigated based on the scenario that rituximab is eligible for reimbursement in Japan as a treatment for ITP. Methods: The efficacy endpoint was set as the number of years with a platelet count ?30 × 109/L. The analysis was conducted from the healthcare payer's perspective. If the first treatment is ineffective or relapse occurs, then the patient is given the following treatment. The analyzed treatment order consisted of three patterns: Splenectomy-romiplostim (sequence 1), splenectomy-romiplostim-rituximab (sequence 2), and splenectomy-rituximab-romiplostim (sequence 3). A Markov model was built for ITP, and the analysis period was set as 2 years. The discount rate was an annual rate of 2%. Sensitivity analyses of the efficacy of splenectomy, romiplostim, and rituximab; treatment cost; and romiplostim dose were performed. Results: The expected costs per patient over a 2-year period for sequences 1, 2, and 3 were USD 40,980, USD 39,822, and USD 33,551, respectively. The expected years with a platelet count ≤30 × 109/L for the three sequences were 1.75, 1.79, and 1.78 years, respectively. The sensitivity analyses illustrated that the results of the base case analysis were robust. Conclusions: Adding rituximab to standard treatment for ITP (sequences 2-3) is less costly and marginally more effective than standard therapy in adults. According to the study results, if rituximab is reimbursed for the treatment of ITP in Japan, medical expenses are expected to decline.

KW - Cost-effectiveness

KW - ITP

KW - Rituximab

KW - Romiplostim

KW - Splenectomy

UR - http://www.scopus.com/inward/record.url?scp=84927937330&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84927937330&partnerID=8YFLogxK

U2 - 10.1186/s12913-015-0681-y

DO - 10.1186/s12913-015-0681-y

M3 - Article

VL - 15

JO - BMC Health Services Research

JF - BMC Health Services Research

SN - 1472-6963

IS - 1

M1 - 2

ER -