PMID- 33828522 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20231102 IS - 1664-2295 (Print) IS - 1664-2295 (Electronic) IS - 1664-2295 (Linking) VI - 12 DP - 2021 TI - The Cost Effectiveness of Immunoglobulin vs. Hematopoietic Stem Cell Transplantation for CIDP. PG - 645263 LID - 10.3389/fneur.2021.645263 [doi] LID - 645263 AB - 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. CI - Copyright (c) 2021 Burt, Tappenden, Balabanov, Han, Quigley, Snowden and Sharrack. FAU - Burt, Richard K AU - Burt RK AD - Division of Immunotherapy, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, United States. FAU - Tappenden, Paul AU - Tappenden P AD - Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Sheffield, United Kingdom. FAU - Balabanov, Roumen AU - Balabanov R AD - Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, United States. FAU - Han, Xiaoqiang AU - Han X AD - Division of Immunotherapy, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, United States. FAU - Quigley, Kathleen AU - Quigley K AD - Division of Immunotherapy, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, United States. FAU - Snowden, John A AU - Snowden JA AD - Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, University of Sheffield, Sheffield, United Kingdom. FAU - Sharrack, Basil AU - Sharrack B AD - Academic Department of Neuroscience and Sheffield, NIHR Translational Neuroscience BRC, Sheffield Teaching Hospitals NHS Foundation Trust, University of Sheffield, Sheffield, United Kingdom. LA - eng PT - Journal Article DEP - 20210322 PL - Switzerland TA - Front Neurol JT - Frontiers in neurology JID - 101546899 PMC - PMC8019941 OTO - NOTNLM OT - CIDP OT - cost OT - health economics OT - hematopoietic stem cell transplantation OT - immunoglobulin COIS- 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. EDAT- 2021/04/09 06:00 MHDA- 2021/04/09 06:01 PMCR- 2021/03/22 CRDT- 2021/04/08 06:26 PHST- 2021/01/14 00:00 [received] PHST- 2021/02/22 00:00 [accepted] PHST- 2021/04/08 06:26 [entrez] PHST- 2021/04/09 06:00 [pubmed] PHST- 2021/04/09 06:01 [medline] PHST- 2021/03/22 00:00 [pmc-release] AID - 10.3389/fneur.2021.645263 [doi] PST - epublish SO - Front Neurol. 2021 Mar 22;12:645263. doi: 10.3389/fneur.2021.645263. eCollection 2021.