INTRODUCTION:Myelodysplastic syndromes/neoplasms (MDS) are a heterogenous group of myeloid cancers that impose a substantial negative impact on patient health-related quality of life. As MDS predominately affects older individuals, who are especially susceptible to the debilitating nature of the disease and its burdensome symptoms, treatment decisions should consider therapeutic value from multiple perspectives to balance clinical efficacy with patient-centered outcomes. This comprehensive approach is known as relative medical value. AREAS COVERED:Although improved outcomes have been observed with hypomethylating agents (HMAs) in patients with higher-risk MDS, parenteral administration of HMA requires frequent infusion clinic visits and is associated with substantial time burden, especially in older patients, impacting treatment persistence. Suboptimal use of HMAs in MDS may lead to poorer outcomes and higher healthcare costs, underscoring the need for patient-centered treatment options that improve persistence. EXPERT OPINION:Oral decitabine and cedazuridine (DEC-C) is an approved treatment for higher-risk MDS and may reduce patient burden associated with parenteral HMA therapy. Oral DEC-C has the potential to improve treatment persistence and clinical outcomes and reduce healthcare resource utilization and costs. This review integrates the available clinical, patient-centered, and economic evidence on the relative medical value of oral DEC-C treatment for MDS.

e23124 Background: Oncology therapeutics are increasingly available via subcutaneous (SC) administration. While previous studies demonstrate a patient preference for SC, there is little real-world research to explain reasons why patients prefer SC over intravenous (IV) administration. Methods: An online survey comprising 44 fixed-response questions and 1 free-text question was conducted from May-June 2024 among US cancer patients (>18 yr) treated with oncology SC therapies having prior IV experience with the same or different drug for the same condition.Qualitative responses of respondents who preferred SC treatment were summarized and thematically analyzed. Individual responses could be coded to more than one theme . Additional analyses were conducted by gender and age. Results: 201 respondents completed the survey [66.17% female; mean age 56.5 (SD = 12.27)]. Participants self-identified as having a blood or solid tumor cancer diagnosis including multiple myeloma (57.71%), breast (34.33%), and non-Hodgkin’s lymphoma (7.46%). Qualitative response analysis of the 180 respondents that preferred SC (89.55%) yielded a total of 233 comments that were grouped into 6 key themes (Table). Gender did not affect response. However, those age >65 view the time impact of treatment administration as significantly more important (40.45%) vs. those < 65 (24.31%). Conclusions: These findings provide insight into the reasons for SC treatment-related preferences of cancer patients. Discussion of administration route effects such as time burden, convenience, tolerability and site of care can inform shared decision-making between providers and patients and may lead to higher quality of care. Key themes. Theme Description Illustrative Quotes % Response (n=233) Time Impact on time related to treatment, travel, personal, work or social activities “Huge life improvement with SC injection. I spend years of my life getting IV medications. I want my time back.” 30.5 Ease & convenience Perception of SC or IV route on treatment experience effort “The injection under the skin is much simpler, you don't need a port that could get infected, you don't need to sit as long.” 27.0 Administration route effects SC or IV administration impacts such as pain, port, infection concerns, tolerability “(SC) is easy to tolerate, I do not get so sick as after infusion, I can do more stuff every day.” 17.6 Travel & location Treatment impacts such as travel, distance, site of care “Injection under the skin is much easier than the port and having to go to the hospital and sit for infusion. It is much more normal to go to the doctor’s office. It seems less like cancer when you go to the doctor’s office.” 13.3 Emotional impact Including confidence, satisfaction, independence “(SC) has given me my life back.” 7.7 Support system Impacts on family or friends “(SC) has made a difference with my caregiver. Gives him more free time and less stress on both of us.” 3.9

34 Background: Trifluridine/tipiracil (FTD/TPI) is a standard of care therapy for patients with refractory metastatic colorectal cancer (mCRC). Recently, FTD/TPI in combination with bevacizumab (BEV) was also approved in this setting based on outcomes from the phase 3 SUNLIGHT trial, including statistically significant improvements in overall survival (OS) compared with FTD/TPI. This study examined demographic/clinical characteristics, treatment duration, OS, and healthcare resource use (HCRU), including outpatient/emergency room (ER) visits and hospitalizations, among patients with mCRC receiving FTD/TPI+BEV versus FTD/TPI in a real-world setting. Methods: This was a retrospective study of adults with CRC, who initiated FTD/TPI as monotherapy or in combination with BEV, identified from the Cerner Enviza electronic health records (EHR) database (12/2020–06/2023). A manual chart review of patients’ medical records was conducted where last follow-up was defined as death or last encounter in the EHR. Descriptive statistics were reported for all study variables; Kaplan Meier curves were used to estimate OS. Results: Of 197 patients abstracted, 122 (62%) received FTD/TPI+BEV combination therapy and 75 (38%) FTD/TPI monotherapy. Mean age was similar between the FTD/TPI+BEV and FTD/TPI groups (60.2 y vs 61.8 y) and 66% vs 63%, respectively, were male. Mean Charlson Comorbidity Index was 8.9 for the FTD/TPI+BEV group vs 8.5 for the FTD/TPI group and body mass index was 26.9 kg/m 2 vs 27.3 kg/m 2 , respectively. Most patients received treatment in the third or fourth line (65% FTD/TPI+BEV; 75% FTD/TPI). Median follow-up was 5.3 and 4.6 mo, respectively. Mean duration of treatment was 3.7 vs 2.7 mo for FTD/TPI+BEV vs FTD/TPI monotherapy (Table). Median OS (from FTD/TPI initiation to death) was 11.5 and 9.6 mo for FTD/TPI+BEV and FTD/TPI groups (number of deaths: 38 and 22), respectively. Patients receiving FTD/TPI+BEV had numerically more outpatient visits (20.5 vs 13.9), but similar ER visits (0.5 each) and slightly fewer hospitalizations (1.1 vs 1.2) compared with FTD/TPI, during follow-up. Overall HCRU costs were similar between groups (27,175 vs 27,891). Conclusions: This real-world study supports the value of FTD/TPI+BEV combination therapy vs FTD/TPI monotherapy as seen in the SUNLIGHT trial. Patients with FTDTPI+BEV were treated for longer duration with improved OS and no difference in trends for HCRU and associated costs. [Table: see text]

WHAT IS THIS SUMMARY ABOUT?:This summary describes the results from a phase 2 study called FOENIXCCA2. The study evaluated treatment with futibatinib in people with a rare form of advanced bile duct cancer called intrahepatic cholangiocarcinoma (or iCCA), where the tumors have changes in the structure of a gene called FGFR2. These changes include FGFR2 gene fusions. Bile duct cancer often returns after surgery or cannot be treated by surgery because the tumor has spread, so it requires treatment with chemotherapy. People live for a median of 1 year after their first chemotherapy treatment and 6 months after their second treatment. This study included people whose cancer had grown/spread after one or more chemotherapy treatments. The aims of the study were to see if futibatinib could shrink the size of tumors and stop the cancer from growing/spreading and to see how long people lived when treated with futibatinib. Clinicians also looked at side effects from taking futibatinib and at how it affected people's quality of life. WHAT WERE THE RESULTS?:Futibatinib treatment shrank tumors in over 80% of people who received treatment. Tumors shrank by at least 30% in 42% of people. Futibatinib stopped tumors from growing/spreading for a median of 9.7 months. People who took the medicine lived for a median of 21.7 months, and 72% of people were still alive after 1 year. Side effects from taking futibatinib were like those reported for similar medicines, and clinicians considered the side effects to be manageable by adjusting the dose of futibatinib or treating the side effects. Most people reported that their quality of life stayed the same or improved during the first 9 months of taking futibatinib. WHAT DO THE RESULTS MEAN?:The results support the use of futibatinib for treating people with advanced bile duct cancer. Based on the results of this study, futibatinib is now approved in the US, Europe, and Japan. Futibatinib is approved for treating adults with advanced bile duct cancer who have received previous treatment for their cancer, and whose tumors have a gene fusion or other change in the FGFR2 gene.Clinical Trial Registration: NCT02052778 (FOENIX-CCA2).

Background: Hypomethylating agents (HMAs) are guideline-recommended treatment for higher-risk myelodysplastic syndromes/neoplasms (MDS). However, a prior survey of patients with MDS reported challenges with intravenous (IV) and subcutaneous (SC) HMA therapies, including pain related to treatment administration and interference with daily activities; most patients also indicated a preference to switch to an oral therapy if one were available. Objectives: This study evaluated the perspectives of US patients with MDS receiving oral decitabine/cedazuridine (DEC-C), an alternative to IV/SC HMAs. Methods: An online survey was conducted among adult patients with MDS in the United States (10 November 2022 to 5 December 2022) who had filled a prescription for oral DEC-C between 2021 and 2022. Results: A total of 150 patients completed the survey; 61% were aged ⩾60 years and 63% were male. Of these, 123 (82%) were still receiving oral DEC-C, and 27 (18%) had stopped oral DEC-C treatment. Half (50%) of patients had received oral DEC-C for ⩾6 months. The majority reported that treatment was convenient (83%) and that they were satisfied with treatment (86%). Most patients also reported very little/no interference with regular daily activities (82%), social activities (78%), and productivity (78%). When queried about negative impacts on quality of life (QOL), treatment side effects were the most commonly reported (30% of respondents). Among patients who had previously received IV/SC HMAs ( n = 91), most agreed that oral DEC-C interfered less with daily life (91%) and had experienced improvement in QOL (85%) compared with previous treatment; 91% reported that oral DEC-C reduced the number of times they needed to travel to a healthcare facility. Conclusion: Survey results suggest very little/no impact on regular daily activities and improved QOL with oral DEC-C relative to IV/SC HMAs, highlighting the potential for oral DEC-C to reduce the treatment burden associated with parenteral HMA therapy.

Introduction: Hypomethylating agents (HMAs) are recommended standard of care for patients with higher-risk myelodysplastic syndromes (HR-MDS). HMA therapies include intravenous (IV) or subcutaneous (SC) decitabine and azacitidine; however, parenteral administration of HMAs has been associated with increased patient burden. The oral HMA decitabine and cedazuridine (DEC-C) was FDA-approved in July 2020 for the treatment of MDS, and offers an alternative to IV/SC HMAs, with similar pharmacokinetic exposure and response rates compared to IV decitabine. This study examined treatment patterns among patients with MDS receiving oral DEC-C compared with IV/SC HMAs in real-world clinical practice. Methods: This was a retrospective analysis using the US Cerner Enviza claims database; medical and prescription claims data for patients were linked to mortality data from Datavant. Adults aged ≥18 years, who were diagnosed with MDS (ICD-10-CM code D46.x; all risk levels), and who had initiated a HMA (≥1 claim; August 1, 2020─August 31, 2022) were included; index date was defined as the first pharmacy or medical claim for DEC-C or IV/SC HMAs, respectively. Patients had variable follow-up after index and were followed until end of enrollment, death, or end of study. Propensity score matching (PSM; 1:1) was performed to balance for confounding factors (age, sex, acute myeloid leukemia [AML] diagnosis, Charlson Comorbidity Index [CCI] score, and red blood cell [RBC] transfusion); adjusted bivariate analysis was used to compare outcomes between treatment groups. Longitudinal persistence was assessed according to the number of cycles of therapy received during follow-up, where a cycle was defined as either 3-10 days of administration of index IV/SC HMA or 1 claim for oral DEC-C, within a 28-day cycle. Results: Of 1569 patients included, 160 (10.2%) received DEC-C and 1409 (89.8%) received IV/SC HMAs. In the DEC-C cohort, median age was 72.5 years and 66.9% were male; in the IV/SC cohort, median age was 69.0 years and 58.2% were male. Two-thirds of patients newly initiated HMAs during the study period (69.4% for DEC-C and 72.2% for IV/SC HMAs); 30.6% of the DEC-C cohort had switched from prior IV/SC HMA treatment. Both groups had high levels of comorbidity; 55.6% of patients in the DEC-C cohort and 58.4% in the IV/SC HMA cohort had CCI scores of ≥3, and 14.4% and 23.8%, respectively, had an AML diagnosis. During the 6-month pre-index period, half (55.3%) of patients in both groups received RBC transfusions, and one quarter (25.9%) received platelet transfusions. After matching, there were 158 patients in each treatment cohort and all covariates included in the PSM were balanced (standardized mean difference <0.2). Median age was 72.0 years and 74.0 years for the DEC-C and IV/SC HMAs matched cohorts, respectively, and 66.5% and 69.6%, respectively, were male. Mean CCI scores were 3.45 for DEC-C and 3.15 for IV/SC HMA groups and 14.6% and 15.8%, respectively, had an AML diagnosis pre index. Longitudinal persistence was comparable between the matched DEC-C and IV/SC cohorts during the first 6 months post-index, with similar proportions receiving the maximum number of treatment cycles (based on a 28-day cycle) at each month following index (73.8% vs 71.1%, 46.5% vs 48.7%, 28.6% vs 24.8%, for 2, 4, 6 months, respectively; Figure). However, a trend towards improved persistence with DEC-C versus IV/SC HMAs was observed in patients receiving treatment beyond 6 months (25.0% vs 17.0%, 18.5% vs 9.0%, 11.4% vs 7.6%, for 8, 10, 12 months, respectively). Mean time to discontinuation of treatment was also numerically higher for DEC-C compared with the IV/SC HMA group (87.7 vs 82.0 days); however, differences were not statistically significant. Conclusions: This study, which is to our knowledge the largest real-world report of oral DEC-C use to date, reveals similar but high levels of disease burden and comorbidities among patients with MDS receiving oral DEC-C and parenteral HMA therapy. Trends in treatment patterns suggest comparable persistence with oral DEC-C and IV/SC HMAs at early stages of therapy and improved persistence with oral DEC-C beyond 6 months. These data support the consideration of oral DEC-C as an alternative to chronic parenteral HMA therapy, with the potential for oral DEC-C to reduce the treatment burden associated with administration of IV/SC HMAs.

BACKGROUND:Alterations in fibroblast growth factor receptor 2 (FGFR2) have emerged as promising drug targets for intrahepatic cholangiocarcinoma, a rare cancer with a poor prognosis. Futibatinib, a next-generation, covalently binding FGFR1-4 inhibitor, has been shown to have both antitumor activity in patients with FGFR-altered tumors and strong preclinical activity against acquired resistance mutations associated with ATP-competitive FGFR inhibitors.METHODS:In this multinational, open-label, single-group, phase 2 study, we enrolled patients with unresectable or metastatic FGFR2 fusion-positive or FGFR2 rearrangement-positive intrahepatic cholangiocarcinoma and disease progression after one or more previous lines of systemic therapy (excluding FGFR inhibitors). The patients received oral futibatinib at a dose of 20 mg once daily in a continuous regimen. The primary end point was objective response (partial or complete response), as assessed by independent central review. Secondary end points included the response duration, progression-free and overall survival, safety, and patient-reported outcomes.RESULTS:Between April 16, 2018, and November 29, 2019, a total of 103 patients were enrolled and received futibatinib. A total of 43 of 103 patients (42%; 95% confidence interval, 32 to 52) had a response, and the median duration of response was 9.7 months. Responses were consistent across patient subgroups, including patients with heavily pretreated disease, older adults, and patients who had co-occurring TP53 mutations. At a median follow-up of 17.1 months, the median progression-free survival was 9.0 months and overall survival was 21.7 months. Common treatment-related grade 3 adverse events were hyperphosphatemia (in 30% of the patients), an increased aspartate aminotransferase level (in 7%), stomatitis (in 6%), and fatigue (in 6%). Treatment-related adverse events led to permanent discontinuation of futibatinib in 2% of the patients. No treatment-related deaths occurred. Quality of life was maintained throughout treatment.CONCLUSIONS:In previously treated patients with FGFR2 fusion or rearrangement-positive intrahepatic cholangiocarcinoma, the use of futibatinib, a covalent FGFR inhibitor, led to measurable clinical benefit. (Funded by Taiho Oncology and Taiho Pharmaceutical; FOENIX-CCA2 ClinicalTrials.gov number, NCT02052778.).

Subcutaneous (SC) administration of rituximab provides an opportunity for reduced patient treatment burden and increased healthcare efficiencies as an alternative to intravenous (IV) rituximab. There is minimal evidence comparing costs associated with SC and IV rituximab in a US setting. This research assessed the impact of transitioning patients from IV to SC rituximab for treatment of non-Hodgkin’s lymphoma (NHL) from the US payer, provider, and patient perspective. We developed a model to estimate cost differences for transitioning 20% of a patient cohort from IV to SC rituximab. We included patients with incident diffuse large B-cell lymphoma, incident and recurrent follicular lymphoma, and incident and recurrent chronic lymphocytic leukemia. In the model, each patient received the same number of doses and that there was no difference in discontinuation between cohorts due to non-inferior efficacy and a similar safety profile. Model inputs were collected from published literature and publicly available data. Scenario analyses tested the impact of availability of low-cost biosimilars. In the base case (1,000,000 covered lives), we estimated a total of 157 patients, with 769 total drug administrations. A transition of 20% of patients from IV to SC was projected to generate $153,000 in payer savings, increase provider capacity by 270 hours, and free 470 hours of patient time. Scenario analyses suggest SC administration will be cost saving for payers even with a market where biosimilars approach 50% market share. A 20% transition to SC rituximab in a single cohort of patients has the potential to generate significant US health system value in the form of payer savings, increased practice capacity, and patient time.

49 Background: Trifluridine/tipiracil (FTD/TPI) and regorafenib are among the limited treatment options in later lines of therapy for patients with advanced colorectal cancer (CRC). While these treatments have demonstrated similar efficacy, differences have been observed in tolerability and data on the impact of such differences on real-world outcomes are limited. To better understand the real world use of FTD/TPI and regorafenib in the US, we report data for adherence/persistence among patients with CRC initiating these therapies. Methods: This was a retrospective cohort study among adults with CRC identified from the IBM MarketScan Commercial Claims and Medicare Supplemental Databases, who initiated FTD/TPI or regorafenib (index) from October 2015-September 2019 and had 6 months of continuous enrollment before the index date. Follow-up was until disenrollment or end of study period. Treatment cohorts were propensity score 1:1 matched, adjusting for differences in socio-demographics, comorbidities, and other baseline characteristics. Adherence and persistence outcomes included time to discontinuation (medication gap of >45 days), medication possession ratio (MPR; number of treated days/ duration of treatment), proportion of days covered (PDC; number of treated days/ days in specified time period), and number of prescriptions received. Results: A total of 1477 patients were included: 892 initiating FTD/TPI (60%) and 585 regorafenib (40%). Demographics were similar prior to matching: mean age was 58 and 59 years, and 57% and 59% were male, in the FTD/TPI and regorafenib groups, respectively. Mean Charlson Comorbidity Index score (excluding cancer) was 0.69 for FTD/TPI initiators and 0.62 for regorafenib initiators. For the matched cohorts (n=585 in each), measures of adherence and persistence (Table) showed longer time to discontinuation (2.5 vs. 2.0 months; p<0.01), and a higher MPR (0.90 vs. 0.87; p<0.01), PDC (at 3 months, 0.74 vs. 0.63; p<0.01), and proportion of patients receiving ≥2 prescriptions (76% vs. 63%; p<0.01) with FTD/TPI vs. regorafenib. Conclusions: Results of this real-world study suggest that patients with CRC initiating FTD/TPI have improved adherence and persistence with therapy compared with those initiating regorafenib. Reasons for this difference and potential impacts on health outcomes require further study. [Table: see text]

Purpose: To depict the treatment journey for patients with small cell lung cancer (SCLC) and evaluate health care resource utilization (HCRU) associated with myelosuppression, a complication induced by chemotherapy or chemotherapy plus radiation therapy. Patients and methods: This was a descriptive, retrospective study of patients with SCLC aged >65 years, identified from linked Surveillance, Epidemiology, and End Results (SEER)-Medicare data curated between January 2012 and December 2015. Treatment types (chemotherapy, radiation therapy, surgery) were classified as first, second, or third line, depending on the temporal sequence in which regimens were prescribed. For each year, the proportions of patients completing 4- or 6-cycle chemotherapy regimens, with hospital admissions associated with myelosuppression, or who used granulocyte colony-stimulating factors (G-CSFs), blood/platelet transfusions, or erythropoiesis-stimulating agents (ESAs), were calculated. Results: Chemotherapy was administered as initial treatment in 7,807/11,907 (65.6%) patients whose treatment journey was recorded. Approximately one-third (n = 3,985) subsequently received radiation therapy. In total, 5,791 (57.8%) patients completed the guideline-recommended 4-6 cycles of chemotherapy. Among all chemotherapy-treated patients, 10,370 (74.3%) experienced >1 inpatient admission associated with myelosuppression (anemia, 7,366 [52.8%]; neutropenia, 4,642 [33.3%]; thrombocytopenia, 2,375 [17.0%]; pancytopenia, 1,983 [14.2%]). Supportive care interventions included G-CSF (6,756 [48.4%] patients), ESAs (1,534 [11.0%]), and transfusions (3,674 [26.3%]). Conclusion: Chemotherapy remains a cornerstone of care for patients with SCLC. Slightly over half of patients completed the recommended number of cycles, underscoring the frailty of patients and aggressiveness of SCLC. HCRU associated with myelosuppression was prominent, suggesting a substantial burden on older patients with SCLC.

e16168 Background: Several fibroblast growth factor receptor (FGFR) inhibitors have been recently approved or are in development for the treatment of cholangiocarcinoma (CCA) with FGFR2 fusions/rearrangements (f/r), such as the ATP-competitive inhibitors pemigatinib and infigratinib (FDA-approved in 2020/2021) and the covalent inhibitor futibatinib, which has shown efficacy in a pivotal phase 2 trial. To inform future evaluations of the impact of these agents in clinical practice, and understand other existing gaps in care, we investigated the characteristics and clinical management of patients with CCA and FGFR2 f/r prior to 2021. Methods: This real-world, retrospective cohort study used de-identified, patient-level electronic health records data from Cancer Treatment Centers of America (CTCA) to identify adults with a diagnosis of advanced intrahepatic CCA (iCCA) who received genomic testing between 2013-2021. Clinical/demographic characteristics, time to genomic testing, treatment patterns, and outcomes of patients with and without FGFR2 f/r were described. Results: 23 patients with FGFR2 f/r and 88 with FGFR wild-type (wt) iCCA were identified. Compared to FGFR wt, FGFR2 f/r had a higher proportion of female (65.2 vs 45.5%) and Black (39.1 vs 18.2%) patients (Table). Mean age at diagnosis was ̃60 y and >60% were diagnosed with stage 4 disease in both cohorts; 91.3% of f/r and 78.4% of wt patients had an ECOG PS 0/1. Median time from diagnosis to genomic testing was ̃5 mo; upon entry to CTCA, median time to testing was <2 mo. 17/23 patients in the f/r cohort had FGFR2 fusions (4 BICC1, 13 other). Three FGFR2 f/r patients received an FGFR inhibitor (pemigatinib) as next treatment after testing in 2020. A trend towards longer median overall survival in the f/r vs wt group (24.5 vs 17.8 mo), and a correlation of survival with ECOG PS were observed. Conclusions: This analysis provides insight into the real-world characteristics, clinical management and outcomes among patients with iCCA and FGFR2 f/r, predominantly prior to the introduction of FGFR inhibitors, providing a baseline for further study as the treatment landscape evolves. Of note, the time lag between diagnosis and genomic testing before entry to CTCA underlines the need for earlier testing to guide optimal therapy in clinical practice. [Table: see text]

Abstract Aims Chemotherapy-induced myelosuppression, which commonly exhibits as neutropenia, anemia, or thrombocytopenia, represents a substantial burden for patients with cancer that affects health-related quality of life and increases healthcare resource utilization (HCRU). We evaluated the burden of myelosuppression among chemotherapy-treated patients with small cell lung cancer (SCLC) using real-world data from community cancer care providers in the Western United States. Materials and methods This was a retrospective, observational analysis of electronic medical records (EMRs) from Providence St. Joseph Health hospital-associated oncology clinics between January 2016 and December 2019. Patient demographics were assessed from the date of first SCLC diagnosis in adult patients with chemotherapy-induced grade ≥3 myelosuppression in first-line (1L) or second-line-and-beyond (2L+) treatment settings. Myelosuppressive adverse events (AEs), treatment patterns, and HCRU were assessed from the date of chemotherapy initiation (index date) until 12 months, date of the last visit, date of death, or study end, whichever occurred earliest. Results Of 347 eligible patients with SCLC who had received chemotherapy (mean age 66; 49% female), all had received at least 1L treatment, and 103 (29.7%) had a 2L + treatment recorded within the EMR during the study period. Of 338 evaluable patients with longitudinal laboratory data, 206 (60.9%) experienced grade ≥3 myelosuppressive AEs, most commonly neutropenia, anemia, and thrombocytopenia (44.9, 41.1, and 25.4 per 100 patients, respectively). Rates of granulocyte colony-stimulating factor use and red blood cell transfusions were 47.0 and 41.7 per 100 patients, respectively. There was a trend toward increasing the use of supportive care interventions and visits to inpatient and outpatient facilities in patients with myelosuppressive AEs in more than one cell lineage. Conclusions Chemotherapy-induced myelosuppression places a substantial real-world burden on patients with SCLC in the community cancer care setting. Innovations to protect bone marrow from chemotherapy-induced damage have the potential to reduce this burden. PLAIN LANGUAGE SUMMARY This study looked at the medical records of people with a particular type of lung cancer known as small cell lung cancer. When treated with chemotherapy, people with this cancer may develop a condition called myelosuppression. This causes people to have fewer blood cells, which can lead to tiredness, or increase the risk of infection or bleeding. The study looked at what types of chemotherapy people with small cell lung cancer were given, what the side effects of myelosuppression were, how often the side effects were reported, and what treatments were given to manage these side effects. The study also looked at whether people with side effects from myelosuppression needed more visits to the doctor or hospital. Around 3 out of 5 people in the study experienced serious side effects resulting in reduced numbers of white blood cells (which fight infection), red blood cells (which carry oxygen), or platelets (which help the blood to clot), and many needed drugs or blood transfusions to treat these side effects. On average, people with side effects from myelosuppression had more visits to healthcare facilities than those people without these side effects. The findings suggest that myelosuppression places a large burden on people with small cell lung cancer who are treated with chemotherapy.

7043 Background: Real-world studies have shown that persistence with intravenous (IV) and subcutaneous (SC) hypomethylating agents (HMAs) among patients (pts) with higher-risk myelodysplastic syndromes (MDS) is poor, with over one-third of treated pts receiving <4 cycles or having a ≥90-day gap in therapy, despite recommendations for at least 4-6 cycles to elicit response in absence of progression/unacceptable toxicity. Survival outcomes have also been shown to be worse, and direct medical costs higher, among HMA non-persistent vs persistent pts. We explored factors associated with early discontinuation of HMA therapy in this population. Methods: This was a retrospective cohort study among pts from the 2010-2016 SEER-Medicare linked database with a diagnosis of refractory anemia with excess blasts (RAEB; a surrogate for higher-risk MDS) from 2011-2015. Included pts had to have received HMA therapy and have ≥12 months’ continuous follow-up after diagnosis. Discontinuation was defined as stopping HMA therapy before 4 cycles. Multivariable logistic regression was used to assess predictors of HMA discontinuation. Results: In total, 664 pts with RAEB and treated with HMAs were included. Overall, 193 (29%) discontinued before 4 cycles; of these, 91 (47%) discontinued after 1 cycle, 57 (30%) 2 cycles, and 45 (23%) 3 cycles. Compared with pts continuing for ≥4 cycles, pts discontinuing before 4 cycles were generally older and more likely to be single/separated/divorced/widowed, have more comorbidities, and have poor performance status (PS) (Table). These trends were most pronounced among pts discontinuing HMA therapy after only 1 cycle vs ≥4 cycles (Table). In multivariable analysis, age 71-75 vs ≥80 y (odds ratio [OR] 0.556, p=0.017) and poor PS (OR 1.585, p=0.019) remained significant predictors of HMA discontinuation. Among treatment-related factors, the most statistically significant association with HMA discontinuation was observed for GCSF use (OR 0.453, p<0.001). Number of pills/day was not a predictor of HMA discontinuation (OR 1.009, p=NS). Conclusions: In this real-world study, almost one-third of RAEB pts treated with IV/SC HMAs discontinued before 4 cycles, with almost half of these pts discontinuing after only 1 cycle. Predictors of HMA discontinuation included older age and poor PS. Novel approaches are needed to improve persistence with HMA therapy, particularly among these higher-risk groups.[Table: see text]

Introduction: Hypomethylating agents (HMAs) are recommended as standard of care treatment for patients with higher-risk myelodysplastic syndromes (MDS); however, intravenous (IV) and subcutaneous (SC) administration of HMA therapy has been associated with additional patient burden. In July 2020, the HMA oral decitabine and cedazuridine (DEC-C) was approved by the US Food and Drug Administration (FDA) for the treatment of MDS, as an alternative to IV or SC HMAs. This study reports initial results evaluating DEC-C and IV/SC HMA treatment patterns and population characteristics among MDS patients in a real-world setting. Methods: This retrospective observational study utilized the IQVIA PharMetrics® Plus database which comprises adjudicated claims for more than 190 million unique patients across the US. Adults with ≥1 claim for HMAs between July 1, 2020 and November 30, 2021, and continuous enrollment for 6-months prior to and ≥1-month following the index date were included. Patients newly initiating HMA therapy were identified, where index was the date of the first claim for DEC-C (DEC-C-new cohort) or IV/SC HMA therapy (IV/SC-new cohort), and patients had no claims for HMA therapy in the prior 6 months; a third cohort including all patients initiating DEC-C as either new HMA therapy or switching from IV/SC HMAs was also identified, where index was the date of first claim for DEC-C (DEC-C-all cohort). Patients in the IV/SC-new cohort also had to have ≥1 diagnosis codes for high-grade MDS lesions and/or refractory anemia with excess blasts. Patients had a variable follow-up period of up to 1-year post-index and were followed through December 31, 2021. Demographic/clinical characteristics and treatment patterns were evaluated across the 3 cohorts according to HMA therapy. Results: Of 208 patients meeting inclusion criteria, 90 initiated DEC-C (DEC-C-all) and 118 initiated IV/SC HMAs (IV/SC-new) during the study period; 59 patients newly initiated HMA therapy with DEC-C (DEC-C-new). Demographic and clinical characteristics were similar across the 3 cohorts (Table), with no statistically significant differences observed between the DEC-C and IV/SC cohorts newly initiating HMA therapy. Overall, the proportion of males was higher (vs females) across the DEC-C-all, DEC-C-new, and IV/SC-new cohorts (61.1%, 62.7%, and 53.4%, respectively), and most patients were aged ≥55 years (93.4%, 91.5%, and 80.4%; median age 65, 67, and 64 years, respectively). All 3 groups had high levels of comorbidity, with mean Charlson Comorbidity Index (CCI) scores of 3.4, 3.3, and 3.1, respectively, and 27.8%, 27.1%, and 24.6% having CCI scores ≥5. Across the DEC-C-all, DEC-C-new, and IV/SC-new groups, common comorbidities included malignancy (58.9%, 55.9%, and 46.6%, respectively), chronic pulmonary disease (25.6%, 27.1%, and 22.9%) and congestive heart failure (20.0%, 15.3%, and 17.8%). In all groups, some patients had a diagnosis of acute myeloid leukemia (AML) during the pre-index period (20.0%, 10.2%, and 21.2%, respectively), and a small proportion received hematopoietic stem cell transplantation (HSCT) pre-index (6.7%, 1.7%, and 1.7%). Red blood cell transfusions were reported in 57.8%, 54.2%, and 53.4%, and platelet transfusions in 28.9%, 25.4%, and 22.9%, for the DEC-C-all, DEC-C-new, and IV/SC-new groups, respectively. Among patients with ≥6-months continuous enrollment post-index, mean number of HMA cycles of therapy (based on number of claims) over the 6-months post-index were similar (4.1, 4.1, and 4.2 cycles in the DEC-C-all, DEC-C-new, and IV/SC-new cohorts, respectively). Conclusions: To our knowledge, this study provides the first real-world data on treatment patterns and characteristics among MDS patients initiating oral DEC-C. Characteristics were similar among patients initiating DEC-C and IV/SC HMAs; data also suggest ongoing off-label use of HMAs post-HSCT transplant and in AML. The similar number of HMA claims between groups suggests comparable compliance with oral therapy at home vs IV/SC treatment in the clinical setting, with a possible advantage of DEC-C in reducing the treatment burden associated with existing IV/SC HMA therapy. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

BACKGROUND COVID-19 is an international health crisis of particular concern in the United States, which saw surges of infections with the lifting of lockdowns and relaxed social distancing. Young adults have proven to be a critical factor for COVID-19 transmission and are an important target of the efforts to contain the pandemic. Scalable digital public health technologies could be deployed to reduce COVID-19 transmission, but their use depends on the willingness of young adults to participate in surveillance. OBJECTIVE The aim of this study is to determine the attitudes of young adults regarding COVID-19 digital surveillance, including which aspects they would accept and which they would not, as well as to determine factors that may be associated with their willingness to participate in digital surveillance. METHODS We conducted an anonymous online survey of young adults aged 18-24 years throughout the United States in June 2020. The questionnaire contained predominantly closed-ended response options with one open-ended question. Descriptive statistics were applied to the data. RESULTS Of 513 young adult respondents, 383 (74.7%) agreed that COVID-19 represents a public health crisis. However, only 231 (45.1%) agreed to actively share their COVID-19 status or symptoms for monitoring and only 171 (33.4%) reported a willingness to allow access to their cell phone for passive location tracking or contact tracing. CONCLUSIONS Despite largely agreeing that COVID-19 represents a serious public health risk, the majority of young adults sampled were reluctant to participate in digital monitoring to manage the pandemic. This was true for both commonly used methods of public health surveillance (such as contact tracing) and novel methods designed to facilitate a return to normal (such as frequent symptom checking through digital apps). This is a potential obstacle to ongoing containment measures (many of which rely on widespread surveillance) and may reflect a need for greater education on the benefits of public health digital surveillance for young adults.

4097 Background: In FOENIX-CCA2 (NCT02052778), a pivotal phase 2 study among iCCA patients (pts) with FGFR2 fusions/rearrangements, the highly selective, irreversible FGFR1–4 inhibitor futibatinib demonstrated a confirmed objective response rate of 41.7%, with a 9.7-month median duration of response. Adverse events were manageable with dosing modifications that did not adversely impact on response. We report outcomes for the preplanned analysis of Patient-Reported Outcomes (PROs) during futibatinib treatment as a secondary objective of FOENIX-CCA2. Methods: Pts enrolled in FOENIX-CCA2 had locally advanced/metastatic unresectable iCCA with FGFR2 fusions/rearrangements, ≥1 prior line of therapy (including gemcitabine/cisplatin) and ECOG PS 0-1. Pts received oral futibatinib 20 mg continuous QD dosing per 21-day cycle. PRO measures included EORTC-QLQ-C30 (1 global health, 5 functional, 9 symptom scales), EQ-5D-3L, and EQ visual analogue scale (VAS). PROs were collected at screening, cycles 2 and 4, every 3 cycles thereafter, and end of treatment. PRO data were evaluated up to cycle 13, the last visit before data were missing for >50% of the PRO population (PRO primary assessment time point). Results: 92/103 (89.3%) pts enrolled had PRO completion data at baseline and a minimum of 1 follow-up assessment (median age 58 y, 56.5% female), with 48 pts having PRO data at cycle 13. At baseline, mean (SD) EORTC QLQ-C30 global health status score was 70.1 (19.4) and EQ VAS score 71.7 (20.3). Mean EORTC QLQ-C30 global health status scores were maintained from baseline to cycle 13, corresponding to 9.0 months on treatment, with no clinically meaningful (≥10-point) changes in individual functional measures (Table). EORTC QLQ-C30 scores across individual symptom measures were also stable from baseline through cycle 13; only constipation showed an average of 10.0-point worsening at only cycle 4. Mean EQ VAS scores were sustained from baseline to cycle 13 (mean change ranging -1.8 to +4.8 across cycles), with values maintained within the population norm range from across 20 countries. Conclusions: Quality of life data from the phase 2 FOENIX-CCA2 trial show that physical, cognitive and emotional functioning, and overall health status were maintained among pts with advanced iCCA receiving futibatinib. Clinical trial information: NCT02052778. [Table: see text]

Studies designed for regulatory approval are necessary but not sufficient for many payer stakeholders. Beyond safety and efficacy data generated within regulatory-purposed randomized controlled trials, payers are concerned with whether the new technology works among more broadly defined 'real world' patients treated under more usual care settings and with comparators reflecting those utilized by their populations (i.e. effectiveness data). In addition, endpoints most important to payers go beyond those in regulatory-purposed trials and include healthcare resource utilization, direct medical care costs, health-related quality of life and increasingly a variety of quality metrics. The pragmatic randomized controlled trial (pRCT) offers a blend of the study rigor required for regulatory approval (e.g., randomization) with the concerns of payers (e.g., broader inclusion criteria, routine practice settings, cost-related endpoints). This chapter delves deeper into these issues and reviews why two recent pRCTs are significant to payers.

Introduction: Hypomethylating agents (HMAs) are standard of care treatment for patients with higher-risk myelodysplastic syndromes (HR-MDS) who are ineligible for stem-cell transplantation or intensive chemotherapy. Until recently, approved HMAs included intravenous or subcutaneous azacitidine and decitabine, which should be administered for a minimum of 4-6 cycles to elicit response in the absence of progression or unacceptable toxicity. In real-world clinical practice, underutilization of HMA therapy has been documented; however, prior estimates have utilized data preceding 2016. The study objectives were to understand recent treatment utilization and characteristics among patients newly diagnosed with HR-MDS in the United States (US).

Myelodysplastic syndromes are hematological malignancies characterized by ineffective hematopoiesis and a high risk of progression to acute myeloid leukemia. Hypomethylating agents (HMAs), azacitidine and decitabine, are standard of care therapy for higher-risk myelodysplastic syndromes. However, outcomes reported for real-world studies fall short of those achieved in clinical trials. We conducted a targeted literature review exploring real-world utilization, persistence and outcomes with intravenous and subcutaneous HMA therapies to better understand barriers to achieving optimal outcomes in clinical practice. The potential benefits of oral HMA therapy were also explored. Underutilization and poor persistence with HMA therapy are associated with suboptimal outcomes, highlighting the need for approaches to improve utilization and persistence, so that patients achieve the optimum benefit from HMA therapy.