Systematic review of costs and cost-effectiveness of treatment for relapsed/refractory acute leukemia in children and young adults

ABSTRACT

Introduction

Survival outcomes of children with relapsed/refractory (r/r) acute leukemia remain poor. Novel expensive treatments have been developed to improve their outcomes, yet, limited evidence exists about cost-effectiveness of alternative treatment strategies.

Areas covered

A systematic review was conducted to summarize health-economic evidence about costs/cost-effectiveness of treating r/r acute leukemia in children/young adults. We searched Medline, Embase, and Cochrane databases until August 13th, 2021. Eligible articles included peer-reviewed original studies addressing r/r pediatric/young-adult acute lymphoblastic leukemia (ALL), and acute myeloid leukemia (AML). Quality assessment was conducted using Consolidated Health Economics Evaluation Reporting Standards (CHEERS) checklist.

Expert Opinion

The majority of papers focused on CAR-T cell therapy, which is still a novel treatment for r/r ALL, and was found to be cost-effective, yet, there remain concerns over its long-term effectiveness, affordability, and equity in access. The next best treatment option is Blinatumomab, followed by Clofarabine therapy, whereas FLA-IDA salvage chemotherapy provides least value for money. The quality of evidence is moderate to high, with limited generalizability of findings due to high variability in outcomes obtained from modeling studies. Limited studies evaluated r/r AML. We provide recommendations to deliver cost-effective treatments in real-world contexts, with implications for healthcare policy and practice.

KEYWORDS:

• Pediatric hematology
• relapsed acute leukemia
• cost-effectiveness
• economic evaluation
• evidence-based medicine
• health policy
• systematic review

Article highlights

• The generated evidence suggests that CAR-T cell therapy is a cost-effective treatment for r/r pediatric ALL compared to other treatment strategies, yet, serious concerns remain over uncertainty of its long-term effectiveness in real-world contexts, affordability of drug price, and equity in access for patients residing in LMICs.

• The next best treatment option is Blinatumomab, followed by Clofarabine therapy, whereas FLA-IDA salvage chemotherapy provided the least value for money.

• There is a gap in knowledge about cost-effectiveness of treatment for r/r Acute Myeloid Leukemia (AML), due to limited published studies.

• Levofloxacin prophylaxis is cost-saving during intensive chemotherapy for r/r acute leukemia.

• The quality of generated evidence is moderate to high, with limited generalizability and applicability of findings due to high variability in outcomes among studies.

Declaration of interest

R Soliman was supported by Egypt Cancer Network (ECN). C Heneghan is funded by the National Institute for Health Research (NIHR) School for Primary Care Research [Project Number 390] and NIHR Oxford BRC. C Heneghan also received expenses for his media work and the WHO. The views expressed are those of the author and not necessarily those of the NIHR, the NHS, or the Department of Health.

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/17474086.2022.2069096

Additional information

Funding

This work was supported by Egypt Cancer Network (ECN); National Institute for Health Research (NIHR) School for Primary Care Research [under Project Number 390].