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. 2021 Mar 22:12:645263.
doi: 10.3389/fneur.2021.645263. eCollection 2021.

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

Richard K Burt  1 , Paul Tappenden  2 , Roumen Balabanov  3 , Xiaoqiang Han  1 , Kathleen Quigley  1 , John A Snowden  4 , Basil Sharrack  5
Affiliations

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

Richard K Burt et al. Front Neurol. .

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.

Keywords: CIDP; cost; health economics; hematopoietic stem cell transplantation; immunoglobulin.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Cost of HSCT in US dollars per CIDP patient. CIDP, chronic inflammatory demyelinating polyradiculoneuropathy; HSCT, hematopoietic stem cell transplantation; US, United States.
Figure 2
Figure 2
Quality of life as measured by the SF-36 rating scale at baseline and for up to 5 years after hematopoietic stem cell transplantation (HCST).
Figure 3
Figure 3
(A) Demonstrates freedom from immunological therapies for up to 5 years after hematopoietic stem cell transplantation (HSCT). (B) Demonstrates reduction in use of ambulatory aids for up to 5 years after HSCT. Replicated with permission from source (16).
See this image and copyright information in PMC

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