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Burden of illness among patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy: a multicenter retrospective chart review study

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Abstract

This study assessed treatment patterns and healthcare resource utilization (HRU) of patients with severe aplastic anemia (SAA) with insufficient response to immunosuppressive therapy (IST). A retrospective chart review was conducted at Dana-Farber Cancer Institute (DFCI), United States, and Hôpital Saint-Louis (HSL), France. Eligible patients were ≥ 18 years old, diagnosed with acquired SAA between January 1, 2006, and July 31, 2016, had insufficient response to IST, and had ≥ 12 months of follow-up post-diagnosis. Overall survival (OS) was estimated using the Kaplan-Meier method. Among the 40 patients, mean age at diagnosis was 44 years and 53% were women. Median follow-up time after SAA diagnosis was 48.3 months. Ninety-five percent of patients received antithymocyte globulin (ATG) as primary therapy prior to hematopoietic stem cell transplant (HSCT). Most common secondary SAA therapies prior to HSCT were eltrombopag (28%) and androgens (15%). Seventy-five percent of patients received HSCT. Prior to HSCT, patients received an average of 2.7 red blood cell (RBC) and 3.3 platelet transfusions per month; patients had 0.9 hospitalizations, 0.4 emergency room visits, and 12.8 office visits per year. Five-year OS was 75%, with infection as the primary cause of death. Additionally, this study provides information on the subgroup of patients receiving eltrombopag which was the most common secondary therapy. This study quantified transfusion and HRU burden associated with SAA and demonstrated high 5-year survival in a recently treated cohort.

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Acknowledgements

The authors would like to thank Yvonne Soohoo from Dana-Farber Cancer Institute and Tiana Andriamasy from Hôpital Saint-Louis for their dedicated support with the data collection. The authors also thank Caroline Korves, ScD, of Analysis Group, Inc. for her assistance with developing this manuscript.

Funding

This research was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ, US.

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Correspondence to Régis Peffault de Latour or Lynn Huynh.

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Conflict of interest

Dr. Huynh, Ms. Ivanova, Mr. Totev, Mr. Bilginsoy, and Dr. Duh are employees of Analysis Group, Inc., a consulting company that has received research funding from Novartis Pharmaceuticals Corporation for this and other studies. Dr. Roy is an employee of Novartis Pharmaceuticals Corporation. Dr. Peffault de Latour and Dr. Antin are employees of institutions, which have received research funding from Novartis Pharmaceuticals Corporation for this study.

Ethical approval

This article does not contain any studies with human participants or animals performed by any of the authors. All study materials were reviewed and approved by local Institutional Review Boards (IRB) in the US (DFCI IRB) and France (CNIL: Commission nationale de l’informatique et des libertés).

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Informed consent was not necessary because retrospectively collected medical records were used in the analysis.

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Peffault de Latour, R., Huynh, L., Ivanova, J.I. et al. Burden of illness among patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy: a multicenter retrospective chart review study. Ann Hematol 99, 743–752 (2020). https://doi.org/10.1007/s00277-019-03809-5

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  • DOI: https://doi.org/10.1007/s00277-019-03809-5

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