Undertreatment of Patients (Pts) with Myelodysplastic Syndromes (MDS): Analysis of a Large Electronic Medical Records (EMR) Database Cohort of US Pts with MDS
Authors: Steensma David P. , Scott Bart L , Ma Xiaomei , Fliss Albert , Kiselev Pavel , Swern Arlene S. , Sugrue Mary M. 
Published: October 2019

Abstract
Introduction: Little is known about current care patterns for pts with MDS across the US with respect to the use of available therapeutic agents. Using a cohort of 5,162 MDS pts we previously identified from the GE Centricity EMR database (GE Healthcare IT, Princeton, NJ) (Ma X, et al. Blood. 2015;126:abstract 3319), we examined associations between pt characteristics and treatment patterns, including sequence of therapies for pts with MDS.
Methods: Pts with data in the EMR from Jan 2006 to end of Feb 2014 were included in this analysis. Pts were grouped by treatment received (erythropoiesis-stimulating agents [ESA], lenalidomide [LEN], hypomethylating agents [HMA; azacitidine or decitabine], and iron chelation therapy [ICT]), either alone, in combination, or as part of a sequence with other therapies. Transfusions were not included in this analysis, as transfusion data were often unrecorded due to transfusions occurring in facilities outside the EMR system. Pt characteristics were evaluated for each treatment group.
Results: Of 5,162 pts evaluated, 1,843 (35.7%) received only 1 therapy, 2,079 (40.3%) received ≥ 1 therapy, with only 236 (4.6%) receiving ≥ 2 therapies. Pts who received ≥ 1 treatment of interest are shown in the Figure. Baseline characteristics for treatment groups are shown in the Table. A total of 85 pts were recorded as having deletion 5q by International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) coding; of these, 66 were recorded as receiving ≥ 1 treatment. Pts were in the EMR system for a median of 29 days (date of first entry to date of last entry), with 36%, 26%, and 15% of pts present in the system for > 6 months, 1 year, and 2 years, respectively.
The most common initial therapy was ESA (n = 1,508; 72.5% of treated pts). Pts treated with ESA first had a median age of 78.0 years; 1,330 (88%) received ESA exclusively. ESA-only pts were the oldest among the treatment groups (median age 79.0 years), had the highest proportion with comorbidities at baseline (69%), and most commonly had isolated anemia. Only a small proportion of pts treated with ESA first subsequently received LEN (n = 79; 5.2%) or HMA (n = 68; 4.5%) as second therapy; median ages of these patients were 76.0 and 73.5 years, respectively.
682 pts (32.7% of treated pts) received a therapy approved specifically for MDS, i.e. HMA and/or LEN, during their treatment.
Pts who received LEN as first treatment (n = 258; 12.4% of treated pts) had a median age of 74.0 years. These pts had a lower median hemoglobin (Hb), lower median absolute neutrophil count (ANC), and similar median platelet count vs pts receiving ESA as first treatment. Most pts who received LEN as their first therapy received it exclusively (244; 94.6% of treated pts); a small number (n = 14) were subsequently treated with HMAs. Pts who received LEN second (n = 99) or third (n = 13) in a sequence of therapies were similar in age (median 76.0 and 74.0 years, respectively) and had similar Hb levels, higher ANCs, and higher platelet counts at baseline than pts who received LEN as first therapy. Most pts (n = 79; 80%) who received LEN as second therapy previously received ESA.
Of 252 pts (12.1% of treated pts) who received HMA as first therapy, 228 (90.4%) received HMA only; median age of patients who received HMAs as first therapy was 75.0 years, and median Hb level, median ANC, and median platelet count were lower than in pts who received ESA as first therapy. Another 100 pts and 28 pts received HMA as second and third therapies, respectively; median age was 73.0 years in each group. Pts receiving HMA third had higher median Hb level, ANC, and platelet count than pts who received HMA as first therapy. Only 61 pts (2.9% of treated pts) received ICT as first therapy.
Conclusions: Pts diagnosed with MDS in the USA are likely to be undertreated. Consistent with findings from physician surveys (e.g. Sekeres M., et al. J Natl Cancer Inst. 2008;100:1542-51), ESAs are the most commonly used therapies despite the lack of a labeled indication for MDS. ESAs are usually the first therapy chosen by physicians and often the only therapy pts with MDS receive. Use of LEN and HMA, which have been approved for the treatment of MDS for ~10 years, appears low in this EMR.

Disclosures
Steensma: Genoptix: Consultancy; Celgene: Consultancy; Millenium/Takeda: Consultancy; Ariad: Equity Ownership; Amgen: Consultancy; Janssen: Consultancy. Scott:Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees. Ma:Celgene Corporation: Consultancy. Fliss:Celgene Corporation: Employment, Equity Ownership. Kiselev:Celgene Corporation: Employment, Equity Ownership. Swern:Celgene: Employment, Equity Ownership. Sugrue:Celgene Corporation: Employment, Equity Ownership.