The Cost Effectiveness of Immunoglobulin vs. Hematopoietic Stem Cell Transplantation for CIDP.

Richard K Burt,John A Snowden,Xiaoqiang Han,Paul Tappenden,Roumen Balabanov,Kathleen Quigley,Basil Sharrack

doi:10.3389/fneur.2021.645263

Abstract

Background: Intravenous immunoglobulin (IVIG) is effective as standard first line therapy for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), but some patients remain dependent on its long-term use. Recently, we have reported that autologous non-myeloablative hematopoietic stem cell transplantation (HSCT) is an effective second line therapy for CIDP.Objectives: To compare the cost of chronic IVIG vs. autologous HSCT (a one-time therapy), we collected data on patients with CIDP undergoing HSCT between 2017 and 2019. This was compared with published literature on the costs and efficacy defined by the Inflammatory Neuropathy Cause And Treatment (INCAT) disability score, Medical Research Council (MRC) sum score, hand grip strength, and SF-36 quality of life (QOL) for CIDP.Methods: Between 2017 and 2019, nineteen patients with chronic CIDP (mean disease treatment duration prior to HSCT of 6 years) underwent autologous HSCT with mean cost of $108,577 per patient (range $56,327–277,119, standard deviation $53,092). After HSCT, 80% of patients remain IVIG and immune treatment free for up to 5 years. In comparison, published cost of IVIG treatment in the USA for an average CIDP patient exceeds $136,000 per year. Despite remaining treatment free, HSCT demonstrated greater improvement in efficacy compared to immunoglobulins.Recommendations: Given the long-term treatment-free remission and better outcome measurements, autologous HSCT is more cost effective than long-term IVIG treatment in patients with chronic CIDP. However, costs will depend on patient selection, the HSCT regimen, and regional variations. Further analysis of the health economics, i.e., cost/outcome ratio, of HSCT as therapy for chronically IVIG dependent CIDP is warranted.

Highlights

chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a demyelination disease of the peripheral nervous system that may pursue a monophasic course of more than 2 months before remitting, follow a relapsing-remitting course, or maintain a chronic progressive course
This is the first report of comparison of costs and health outcomes for hematopoietic stem cell transplantation (HSCT) compared to chronic Intravenous immunoglobulin (IVIG) that, over a 5-year interval and in properly selected patients, appears to result in a health care savings of ∼500,000.00 US dollars per patient
It appears that when using a non-myeloablative regimen in properly selected patients that HSCT may be performed safely, could achieve a substantial cost savings for private or public insurance, and give superior results such as improvement in NCS and QOL compared to continued IVIG

Summary

Introduction

CIDP is a demyelination disease of the peripheral nervous system that may pursue a monophasic course of more than 2 months before remitting, follow a relapsing-remitting course, or maintain a chronic progressive course. First line therapies are corticosteroids, IVIG [3, 4], subcutaneous immunoglobulin (SCIg) [5], and plasmapheresis (PLEX) [6]. Some patients fail to respond to first-line treatments [7] or respond but become dependent on their longterm use [8]. IVIG and SCIg are expensive, especially when required for chronic long-term treatment, restrict quality of life, and may have significant side effects including anaphylaxis, aseptic meningitis, and maintenance of and complications from chronic intravenous access. Intravenous immunoglobulin (IVIG) is effective as standard first line therapy for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), but some patients remain dependent on its long-term use. We have reported that autologous non-myeloablative hematopoietic stem cell transplantation (HSCT) is an effective second line therapy for CIDP

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