The Incidence and Health Care Resource Burden of the Myelodysplastic Syndromes in Patients in Whom First-Line Hypomethylating Agents Fail

Cogle, Christopher R.; Kurtin, Sandra; Bentley, Tanya G. K.; Broder, Michael S.; Chang, Eunice; Megaffin, Scott; Fruchtman, Steven; Petrone, Michael E.; Mukherjee, Sudipto

doi:10.1634/theoncologist.2016-0211

Cited by 17 publications

(16 citation statements)

References 16 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Without doubt, MDS patients for whom HMA therapy fails are a population with an extremely dismal outcome and with few therapeutic opportunities, whose difficult care burdens the health system. 85 Proper management of first-line HMA therapy, with appropriate doses and prolonged treatment, may partially reduce primary resistance. Eventually, HMA-treated MDS patients lose response, and the best treatment to offer at present is inclusion in experimental trials.…”

Section: Discussionmentioning

confidence: 99%

How I treat MDS after hypomethylating agent failure

Santini

2019

Blood

View full text Add to dashboard Cite

Hypomethylating agents (HMA) azacitidine and decitabine are standard of care for myelodysplastic syndrome (MDS). Response to these agents occurs in ∼50% of treated patients, and duration of response, although variable, is transient. Prediction of response to HMAs is possible with clinical and molecular parameters, but alternative approved treatments are not available, and in the case of HMA failure, there are no standard therapeutic opportunities. It is important to develop a reasoned choice of therapy after HMA failure. This choice should be based on evaluation of type of resistance (primary vs secondary, progression of disease [acute leukemia or higher risk MDS] vs absence of hematological improvement) as well as on molecular and cytogenetic characteristics reassessed at the moment of HMA failure. Rescue strategies may include stem-cell transplantation, which remains the only curative option, and chemotherapy, both of which are feasible in only a minority of cases, and experimental agents. Patients experiencing HMA failure should be recruited to clinical experimental trials as often as possible. Several novel agents with different mechanisms of action are currently being tested in this setting. Drugs targeting molecular alterations (IDH2 mutations, spliceosome gene mutations) or altered signaling pathways (BCL2 inhibitors) seem to be the most promising.

show abstract

Section: Discussionmentioning

confidence: 99%

How I treat MDS after hypomethylating agent failure

Santini

2019

Blood

View full text Add to dashboard Cite

show abstract

“…To our knowledge, no other comparisons of HRU outcomes in HMA persistent versus non-persistent patients have been conducted. However, a small number of studies have assessed HRU in patients after HMA therapy failure and found the burden to be substantial [15,16]. For instance, in one study [16], MDS patients had 25.9 all-cause physician office visits on average during the 6 months after first-line HMA failure (i.e.…”

Section: Discussionmentioning

confidence: 99%

“…However, a small number of studies have assessed HRU in patients after HMA therapy failure and found the burden to be substantial [15,16]. For instance, in one study [16], MDS patients had 25.9 all-cause physician office visits on average during the 6 months after first-line HMA failure (i.e. ∼4.3 visits per month), with 32.6% and 19.7% of patients having at least 1 all-cause IP and ED visit, respectively.…”

Section: Discussionmentioning

confidence: 99%

“…The HRU burden associated with HMAs is likely further magnified among MDS patients who are not persistent to treatment, given that non-persistence is a known risk factor for worse health outcomes and increased economic burden [12,16,17]. Indeed, two retrospective cohort studies reported a substantial HRU burden among MDS patients after 1st-line HMA failure [15,16]. However, to date, no studies have compared HRU outcomes among HMA persistent versus non-persistent patients.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Persistence to hypomethylating agents and clinical and economic outcomes among patients with myelodysplastic syndromes

et al. 2021

View full text Add to dashboard Cite

Objectives: To evaluate hypomethylating agent (HMA) persistence in patients with myelodysplastic syndromes (MDS), and examine its association with healthcare resource utilization (HRU) and progression to acute myeloid leukemia (AML). Methods: A total of 2,400 adults diagnosed with MDS initiating HMAs were included from IBM MarketScan databases during 1/1/2011-3/31/2018. The index date was HMA initiation following MDS diagnosis. Patients were classified according to their persistence status by the end of a fixed 'landmark period' of 4 months post-index. Results: Median persistence to HMAs was 5.6 months (95% CI: 5.2, 6.1); HMA non-persistence increased with time. Non-persistent patients had a significantly higher non-HMA-related HRU burden than persistent patients [adjusted incidence rate ratios, outpatient visits: 1.12 (95% CI: 1.10, 1.14); inpatient visits: 1.48 (95% CI: 1.30, 1.69); emergency department visits 1.30 (95% CI: 1.12, 1.50); all p-values < 0.001]. All-cause and HMA-related outpatient visits were lower among non-persistent patients, likely because of fewer HMA administration-related visits. The incidence rate of AML was numerically, although not significantly, higher in non-persistent patients, when starting follow-up at the end of the landmark period. When follow-up began at the index date, non-persistent patients had a significantly higher rate of AML [adjusted hazard ratio, 1.88 (95% CI: 1.53, 2.32); p-value < 0.001]. Conclusions: HMA non-persistence, which increased over time, was associated with significantly higher non-HMA-related HRU, and numerically higher AML progression in MDS patients initiating HMAs. Future studies should evaluate predictors of HMA non-persistence in this patient population.

show abstract

“…The mean total healthcare costs were estimated to be $17,361 perpatient-per-month (PPPM; 2015 US dollars [USD]) in higherrisk MDS patients receiving MDS-related treatment in commercial data, and costs for patients who progressed to AML or died were twice those observed in patients without evidence of progression or death 20 . Moreover, another study assessed costs related to MDS treatment failure in patients using HMA and estimated that the mean MDS/AML-related total cost of care was $76,945 during the first 6 months postfailure from 2009 to 2012 21 .…”

Section: Introductionmentioning

confidence: 99%

Healthcare resource utilization and costs in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis

Stein

Bonifacio

Latrémouille-Viau

et al. 2021

Journal of Medical Economics

View full text Add to dashboard Cite

Aims: To describe healthcare resource utilization (HRU) and costs in patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA) based on HMA-treatment response. Materials and methods: SEER-Medicare data (January 2006-December 2016) were used to identify adults diagnosed with MDS (SEER: January 2009-December 2015) initiated on HMA (index date). HMAtreatment success (indicators: 7 HMA cycles, stem cell transplantation, and transfusion independence) or failure (indicators: acute myeloid leukemia [AML], AML-like treatment, and death) was determined using a claim-based algorithm. HRU and costs were assessed from the index date to 1-year post-index, overall and stratified by HMA-treatment success or failure. Among patients with HMA-treatment failure, HRU and costs were also assessed from failure to 1-year post-failure.Results: The study included 3,046 patients (mean age: 77.4 years; females: 36.8%). Rates of HMA-treatment success and failure were 44.4% and 76.2%, respectively (20.6% had HMA-treatment success then failure). Overall, patients had 15.2 inpatient admissions per-100-patients-per-month (median follow-up: 5.9 months). Patients with HMA-treatment success had 7.5 inpatient admissions per-100-patients-permonth (median follow-up: 12.0 months), while those with HMA-treatment failure had 20.4 and 35.3 admissions per-100-patients-per-month pre-and post-HMA-treatment failure, respectively (median follow-up: 4.3 and 1.8 months, pre-and post-HMA-treatment failure, respectively). Mean total healthcare costs were $12,494 per-patient-per-month overall, $8,069 per-patient-per-month among patients with HMA-treatment success, and $13,809 and $19,242 per-patient-per-month pre-and post-HMA-treatment failure, respectively. Outpatient costs (68.3%) were the main contributor of total healthcare costs overall, while inpatient costs (80.3%) were the main cost driver post-HMA-treatment failure. Limitations: Without available laboratory test results, clinical indicators observed in claims were used to assess HMA-treatment response. Conclusions: Over 75% of patients with MDS failed HMA-treatment within 6 months of initiation and were observed with more inpatient admissions than those with HMA-treatment success, translating into substantially higher healthcare costs. HMA-treatment failure results in an important economic burden in MDS patients.

show abstract

The Incidence and Health Care Resource Burden of the Myelodysplastic Syndromes in Patients in Whom First-Line Hypomethylating Agents Fail

Cited by 17 publications

References 16 publications

How I treat MDS after hypomethylating agent failure

How I treat MDS after hypomethylating agent failure

Persistence to hypomethylating agents and clinical and economic outcomes among patients with myelodysplastic syndromes

Healthcare resource utilization and costs in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis

Contact Info

Product

Resources

About