Burden Of Illness In Patients Research Articles

Impact of Social Needs and Identity Experiences on the Burden of Illness for Patients with Multiple Myeloma: A Mixed-Methods Study

Impact of Social Needs and Identity Experiences on the Burden of Illness for Patients with Multiple Myeloma: A Mixed-Methods Study

MM-416 Impact of Social Needs and Identity Experiences on the Burden of Illness for Patients with Multiple Myeloma: A Mixed-Methods Study

MM-416 Impact of Social Needs and Identity Experiences on the Burden of Illness for Patients with Multiple Myeloma: A Mixed-Methods Study

Impact of Social Needs and Identity Experiences on the Burden of Illness in Patients with Multiple Myeloma: A Mixed-Methods Study.

Multiple myeloma (MM) is a common hematologic malignancy, but due to its incurable nature, patients experience many relapses in their lifetime and hence face unique challenges. This mixed-methods study consisting of an online survey and subsequent focus groups aimed to understand how social and identity experiences affected the diagnostic, treatment, and care journey for patients with MM. Twenty-three adult patients with MM participated in this study. The survey participants identified common determinants negatively impacting their health, including mental health concerns (experienced by 90.5% of respondents), worries about food shortage (42.9%), and transportation concerns (28.6%). Focus group participants described high physical and mental health burdens associated with MM. Frequent monitoring, fear of a relapse, and unpredictable side effects contributed to high anxiety. Participants indicated that MM differed from other types of cancer and chronic health conditions in many ways, particularly how and where the diagnosis was made, disease progression and relapse, treatments and side effects, and financial concerns. Most participants (65.0%) reported ≥1 social need that negatively impacted health outcomes including lack of knowledge about MM, financial instability, and lack of insurance, transportation, and social support. The findings reveal that patients with MM continually experience patient-specific mental and physical health burdens indicating high unmet needs throughout the disease journey.

Burden of illness in patients with pulmonary hypertension due to interstitial lung disease: a real-world analysis

BackgroundPulmonary hypertension due to interstitial lung disease (PH-ILD) is associated with high rates of respiratory failure and death. Healthcare resource utilization (HCRU) and cost data are needed to characterize PH-ILD disease burden.MethodsA retrospective cohort analysis of the Truven Health MarketScan® Commercial Claims and Encounters Database and Medicare Supplemental Database between June 2015 to June 2019 was conducted. Patients with ILD were identified and indexed based on their first claim with a PH diagnosis. Patients were required to be 18 years of age on the index date and continuously enrolled for 12-months pre- and post-index. Patients were excluded for having a PH diagnosis prior to ILD diagnosis or the presence of other non-ILD, PH-associated conditions. Treatment patterns, HCRU, and healthcare costs were compared between the 12 months pre- versus 12 months post-index date.ResultsIn total, 122 patients with PH-ILD were included (mean [SD] age, 63.7 [16.6] years; female, 64.8%). The same medication classes were most frequently used both pre- and post-index (corticosteroids: pre-index 43.4%, post-index 53.5%; calcium channel blockers: 25.4%, 36.9%; oxygen: 12.3%, 25.4%). All-cause hospitalizations increased 2-fold, with 29.5% of patients hospitalized pre-index vs. 59.0% post-index (P < 0.0001). Intensive care unit (ICU) utilization increased from 6.6 to 17.2% (P = 0.0433). Mean inpatient visits increased from 0.5 (SD, 0.9) to 1.1 (1.3) (P < 0.0001); length of stay (days) increased from 5.4 (5.9) to 7.5 (11.6) (P < 0.0001); bed days from 2.5 (6.6) to 8.0 (16.3) (P < 0.0001); ICU days from 3.8 (2.3) to 7.0 (13.2) (P = 0.0362); and outpatient visits from 24.5 (16.8) to 32.9 (21.8) (P < 0.0001). Mean (SD) total all-cause healthcare costs increased from $43,201 ($98,604) pre-index to $108,387 ($190,673) post-index (P < 0.0001); this was largely driven by hospitalizations (which increased from a mean [SD] of $13,133 [$28,752] to $63,218 [$75,639] [P < 0.0001]) and outpatient costs ($16,150 [$75,639] to $25,604 [$93,964] [P < 0.0001]).ConclusionPH-ILD contributes to a high HCRU and cost burden. Timely identification, management, and treatment are needed to mitigate the clinical and economic consequences of PH-ILD development and progression.

Individual Burden of Illness Index in Bipolar Disorder Remission: A Cross-Sectional Study.

A population-based method for estimating disease burden is commonly used. Nevertheless, these measurements do not entirely capture the comprehensive burden of illness on an individual patient. To address the problem, the Individual Burden of Illness Index (IBI index) Index was created and validated, specifically for major depressive disorder. The IBI represents the overall influence of the condition, encompassing distress from symptom intensity, functional impairment, and the patient's quality of life. The aim of the study was to approve and validate the IBI index for the integral assessment of disease burden in patients with bipolar disorder (BD) in remission. The cross-sectional study was conducted in the outpatient psychiatric services in Saint Petersburg, Russia, from April through October 2020. Eighty-five patients aged 18 to 45 (mean age 36.6±5.7 years) with BD (type I - 75%, n=64; type II - 25%, n=21) in remission were examined. The study procedure included a structured clinical interview and the use of clinical scales: the World Health Organization's Quality of Life Questionnaire, Hamilton Rating Scale for Depression (HDRS), Young Mania Rating Scale (YMRS), and Personal and the Social Performance Scale. The principal component analysis in accordance with the adjusted one showed that the burden of illness in patients with BD in remission is directly related to the severity of residual depressive symptoms, reflected in the HDRS score: as the HDRS score increases (0.27, p <0.001), residual mania (-0.14, p <0.001), social functioning (-0.06, p <0.001), and quality of life (-0.04, p <0.001) decrease. In contrast, when there are remaining residual mania symptoms, as indicated by the YMRS score, the result tends to be a lower burden, better social functioning, and enhanced quality of life. The study has demonstrated through statistical means a successful adaptation and validation of the previously calculated IBI index for patients with BD in remission. Residual affective symptoms were shown to have different impacts on the social functioning of patients with BD in remission, indicating the need for a timely assessment and targeted therapy of these symptoms in such patients.

Retrospective Database Analysis of Liver-Related Clinical Events in Adult and Pediatric Patients with Alpha-1 Antitrypsin Deficiency in the United States.

Real-world analyses on burden of illness in patients with alpha-1 antitrypsin deficiency (AATD) are limited. We investigated the real-world burden of liver-related clinical events among adult and pediatric patients with AATD in the USA. This was a retrospective, observational analysis of administrative claims data from the IQVIA PharMetrics® Plus and Ambulatory Electronic Medical Records databases from 2011 to 2022. Patients had a diagnosis of liver and/or lung disease with ≥180 days of continuous enrollment in the IQVIA PharMetrics Plus database before and ≥90 days after their first diagnosis. Follow-up time was assigned to the AATD with liver disease health state or AATD with both liver and lung disease health state (for patients aged ≥18 years only). Baseline demographic characteristics and liver-related clinical events of interest were reported. Of 5136 eligible patients, 771 adult and 123 pediatric patients contributed time to the AATD with liver disease health state; 541 adults contributed time to the AATD with both liver and lung disease health state. Among adults, patients with both liver and lung disease had higher rates of liver-related clinical events than patients with liver disease alone. Ascites was the most frequently observed clinical event among adults in both health states, and the median time to the composite of any liver-related clinical event was 26.5 days among all adults combined. Across all pediatric age groups, ascites, gastrointestinal bleed and hepatic encephalopathy were more common than spontaneous bacterial peritonitis and hepatocellular carcinoma, but median time to liver-related clinical event varied by age group at index date and type of event. No liver transplantations occurred in patients aged 6-17 years. Diagnosed AATD with liver disease carries a substantial burden on adult and pediatric patients; new treatment options are warranted to avoid disease progression to decompensating events.

The Assessment of Clinical Characteristics, Treatment Patterns, and Burden of Illness in Patients With Episodic and Chronic Migraine: A Cross-Sectional Study.

Migraineis a prevalent and disabling primary headache disorder worldwide, causing significant years lost due to disability (YLD) and impacting various aspects ofeveryday life. Despite its high prevalence and substantial burden, there is a lack of comprehensive data on clinical patterns and management trends,in places like Tamil Nadu, India. This study aims and also fill gaps by investigating and analyzing the clinical characteristics, treatment patterns, and illness burden among patients with episodic migraine (EM) and chronic migraine (CM) in the state of Tamil Nadu. This cross-sectional retrospective study was conducted at the Department of Neurology, Madras Medical College, Chennai, over a three-month period starting from January 2024 to March 2024. The study included migraine patients aged 18 years and above who met theInternational Classification of Headache Disorders (ICHD)-3 criteria and took treatment at the department. Data were collected using patient interviews, medical records, and counseling sessions and using a pre-designed questionnaire. Patient demographics, clinical characteristics, symptom prevalence, prescription patterns, and illness burden were analyzed accordingly. The Migraine Disability Assessment (MIDAS) questionnaire was used to measure the burden of illness. The analysis involved 400 migraine patients, 92.5% of them having EM and 7.5% of them having CM. The mean age of patients was 37.5 years, with a predominance of females (73.5%). Patients with CM had having significantly higher average number of headache days per month when compared to those with EM. Tension-type headache (TTH) and medication-overuse headache (MOH) were more prevalent in those CM patients. Trigger factors includelack of sleep, bright light exposure, and stress. Comorbidities such as diabetes mellitus, obesity, and depression were significantly higher in CM patients. Acute treatment included NSAIDs and Triptans, while preventive therapy was more commonly used in CM patients. The mean MIDAS score was significantly higher in CM patients, which indicates greater disability. The study provides valuable insights into the clinical characteristics, treatment patterns, and burden of illness among migraine patients in Tamil Nadu, India. Significant differences were observed between EM and CM patients, which highlights the need for comprehensive management strategies. Preventive therapy, lifestyle modifications, and comprehensive assessment of disability are all important in addressing the variable needs of migraine patients and also reducing theburden of illness. Further research is necessary to explore additional factors influencing migraine outcomes in this population.

Clinical and Economic Burden of Illness in Patients with Persistent or Chronic Primary Immune Thrombocytopenia Treated with TPO-Ras and Rituximab

Background Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by reduced platelet counts. Patients with ITP lasting for ≥3 months (persistent or chronic primary ITP), and who do not respond adequately or maintain a response to initial therapy, are usually treated with advanced therapies such as thrombopoietin receptor agonists (TPO-RAs [avatrombopag/romiplostim/eltrombopag]), rituximab, and fostamatinib. The economic and clinical outcomes among patients on advanced therapies for ITP are not well described in real-world data. Objective This study aimed to evaluate the clinical and economic burden among ITP patients (lasting ≥3 months) treated with TPO-RAs, or rituximab, or fostamatinib by therapy type. Methods This retrospective cohort study used the US administrative health insurance claims data from the Optum's de-identified Clinformatics ® Data Mart Database. Patients with ITP lasting ≥3 months (aged ≥18 years), treated with TPO-RAs, or rituximab, or fostamatinib (index) between October 1, 2015, to August 31, 2020, after a 180-days washout period were included. Patient characteristics were evaluated 180 days prior to index (baseline). Follow-up for clinical outcomes started one-day after the index date and on the index date for economic outcomes. Patients were censored on switch to another ITP therapy different from index, death, end of data, or end of enrollment. Clinical outcomes (bleeding events [overall and type of bleeding], thromboembolic events [TEs], and clinical events identified as potential drug related adverse reactions) were reported as rate of events per 1000 person-years (PY). Health care resource use included number of hospitalizations, hospital days, emergency room visits, number of intensive care unit (ICU) admissions (both reported as rate of events per 1000 PY), and length of ICU stay. All analyses were stratified by treatment type on index date, however formal adjusted comparisons were not performed. Outcomes for fostamatinib users are not presented due to low sample size. Results A total of 404 patients treated with TPO-RAs, 333 with rituximab, and 14 with fostamatinib were eligible for the analysis. The average age of patients across the three cohorts was 60 years; 57.0% were females. The most common comorbidities across the three cohorts were diabetes (26.8%), coronary artery disease (14.4%), chronic heart failure (9.3%), and any TE (10.5%). At baseline, 29.0% of TPO-RA users, 30.6% of rituximab users, and 28.6% of fostamatinib users had experienced bleeding events. At baseline, bacterial infections occurred in 2% to 4% of patients treated with TPO-RAs and rituximab. At baseline, 36.1% TPO-RA users, 52.9% rituximab users, and 42.9% fostamatinib users had required any rescue therapy (IV anti-D, IV immunoglobulins, IV steroids, and platelet transfusion). Patients initiating TPO-RAs and rituximab were observed for a median (interquartile range, IQR) duration of 629 (286-1077) days and 398 (75-991) days, respectively. TPO-RA users and rituximab users stayed on the therapy for a mean (SD) duration of 305 (356) days and 31 (20) days, respectively. Among rituximab users, 60.7% completed a 4-week treatment course at index. During follow-up, the rate of bleeding events among TPO-RA and rituximab users was 284 and 254 per 1000 PY, respectively. The most common bleeding events were gastrointestinal bleeding, hematuria, and epistaxis. The rate of bacterial infections was 71 per 1000 PY in patients treated with TPO-RAs and 58 per 1000 PY in those treated with rituximab. The rate of TEs among TPO-RA and rituximab users was 153 and 110 per 1000 PY, respectively (Table 1). During follow-up, 38.4% of TPO-RA users and 55.9% of rituximab users required rescue therapy. The rate of hospitalizations among patients treated with TPO-RAs and rituximab was 573 and 400 per 1000 PY, respectively. The rate of ICU stays in patients treated with TPO-RAs was 176 per 1000 person-years, and 143 per 1000 person-years for those treated with rituximab (Table 2). Conclusion ITP patients (lasting ≥ 3 months) treated with TPO-RAs and rituximab continue to experience substantial rates of infections, TEs and bleeding (often requiring rescue therapies). All-cause hospitalization was high in this population. Further studies are needed to understand long-term clinical and economic outcomes among ITP patients.

1865. Refractory Nontuberculous Mycobacterial Pulmonary Disease: Natural History and Burden of Illness in Patients from the US Bronchiectasis and NTM Research Registry (BRR)

Abstract Background Nontuberculous mycobacterial pulmonary disease (NTMPD) is a chronic lung infection that is challenging to treat, especially refractory NTMPD. Data are limited on the natural history, risk profile, and burden of refractory NTMPD. The objective of this study was to evaluate the burden of illness (BOI) among patients with NTMPD who later develop refractory NTMPD vs those who do not. Methods This was a retrospective analysis using BRR data. Patients treated for NTMPD were included and stratified as refractory (received oral clofazimine/bedaquiline or inhaled amikacin/ALIS, or remained sputum culture positive despite ≥ 6 months of treatment) or nonrefractory. Modified bronchiectasis severity index (mBSI) at baseline visit and BOI including comorbid conditions and surgeries at the treatment visit were compared by cross-sectional analysis between the two groups. Longitudinal analysis of BOI focused on exacerbations and hospitalizations in the refractory group at their pre-refractory visit and refractory visit (first visit meeting the refractory definition). Results A total of 1,064 patients treated for NTMPD were included: 462 (43.4%) refractory and 602 (56.6%) nonrefractory. During the baseline period, the refractory group had more severe bronchiectasis (Table 1, mBSI, mean [SD], 7.3 [3.4] vs 6.7 [3.4]). At the time of treatment, bronchiectasis was more common in the refractory than nonrefractory (96.2% vs 89.7%) group, as were hemoptysis (26.6% vs 16.3%) and GERD (50.5% vs 39.9%). Except BMI, there were no statistically significant differences between the pre-refractory and nonrefractory treatment visits (Table 2). In the refractory group, exacerbations and hospitalizations remained high from the pre-refractory to refractory visit (45.6% vs 39.4% and 13.3% vs 12.8%, respectively). Oxygen and bronchial hygiene use increased from the pre-refractory to the refractory visit (5.2% vs 9.3% and 54.5% vs 61.7%, respectively) consistent with increased management of respiratory conditions when not responding to treatment (Table 2). Conclusion Refractory NTMPD imposes a high disease burden on patients in the BRR. This high burden was present at pre-refractory visits and through the development of refractory status. Disclosures Timothy R. Aksamit, MD, AstraZeneca: Advisor/Consultant|Baxter International, Hill-Rom: Advisor/Consultant|Insmed Incorporated: Advisor/Consultant|Johnson &amp; Johnson: Advisor/Consultant|Redhill Biopharma: Advisor/Consultant|RespirTech: Advisor/Consultant|Spero Therapeutics: Advisor/Consultant|Zambon: Advisor/Consultant David M. Mannino, MD, AstraZeneca: Advisor/Consultant|GlaxoSmithKline: Advisor/Consultant|Up to Date: Advisor/Consultant Ping Wang, PhD, Insmed Incorporated: Salary|Insmed Incorporated: Stocks/Bonds Mariam Hassan, PhD, B. Pharm, Insmed Incorporated: Salary|Insmed Incorporated: Stocks/Bonds

EE539 Estimating the Lifetime Burden of Illness in Patients with Bronchiolitis Obliterans Syndrome (BOS)

Bronchiolitis Obliterans Syndrome (BOS) is a rare, life-threatening disease where the immune system attacks the airways of the lungs. BOS is estimated to affect approximately 25,000 individuals worldwide and can occur after hematopoietic stem cell transplantation (HSCT) or lung transplantation, with up to half of all lung transplant recipients developing BOS within 5 years of transplantation. BOS is rapidly progressive and irreversible, leading to lung failure after diagnosis, and making it the leading cause of death. The aim of this analysis was to estimate the lifetime economic burden of illness for BOS patients from the perspective of a US Payer.

HTA64 Burden of Illness in Patients with Facial Angiofibroma Associated with Tuberous Sclerosis Complex: A Systematic Review

This systematic literature review assessed the epidemiological, humanistic and economic burden of facial angiofibroma (FA) associated with tuberous sclerosis complex (TSC), to identify evidence and data gaps.

Comparing the burden of illness in patients with alopecia areata vs atopic dermatitis in the US population from a payer perspective.

BACKGROUND: Alopecia areata (AA) is an autoimmune disease characterized by nonscarring hair loss. AA frequently co-occurs with other inflammatory autoimmune conditions, presenting a significant clinical burden. OBJECTIVE: To compare the burden of illness, direct and indirect costs in adult patients with AA vs atopic dermatitis (AD). METHODS: This retrospective cohort study used US administrative claims data from the Merative MarketScan Commercial Claims and Encounters Database to compare commercially insured adults with AA to those with AD. Patients with an AA diagnosis between January 2017 and September 2019 were propensity score matched to patients with AD. Comorbidity burden, medication use, health care resource utilization, health care costs, and indirect costs during a 12-month follow-up period were compared between cohorts. RESULTS: Overall, 25,446 adult patients with AA were selected for the matched analysis with the AD cohort. Patients with AA generally had lower comorbidity burden than patients with AD; mean Deyo-Charlson Comorbidity Index scores were 0.36 (SD = 0.99) and 0.39 (SD = 0.92), for AA and AD, respectively (P = 0.007). Patients with AA had significantly lower proportions of allergic rhinitis, asthma, pruritus, skin infections, and urticaria, but higher proportions of thyroid disease, when compared with patients with AD (all P < 0.001). A smaller proportion of patients with AA had prescriptions for topical (45.3% vs 64.8%; P < 0.001) and oral (20.3% vs 29.6%; P < 0.001) corticosteroids and antianxiety and/or antidepressants (24.7% vs 29.7%; P < 0.001), but a significantly larger proportion for intralesional corticosteroids (triamcinolone) (49.6% vs 21.7%; P < 0.001), compared with patients with AD. Despite a lower comorbidity burden and generally less medication usage in patients with AA, total all-cause health care costs did not significantly differ between the AA and AD cohorts ($10,705 vs $10,816; P = 0.712), and outpatient costs were higher in patients with AA ($6,297 vs $5,859; P = 0.014). Female patients with AA had significantly greater costs for both outpatient and outpatient pharmacy when compared with female patients with AD. Patients with AA were more likely to have a claim for long-term disability (0.6% vs 0.3%; P = 0.001) and higher long-term disability-associated indirect costs ($73 [SD = $1,442] vs $25 [SD = $774]; P = 0.004) compared with patients with AD. CONCLUSIONS: We found similar total health care costs in patients with AA and AD, despite a lower proportion of comorbidities and prescription use in patients with AA. Outpatient costs were also significantly higher overall in patients with AA. Although often dismissed as a cosmetic condition, AA, an autoimmune disease, has a similar level of medical expenditure as AD. DISCLOSURES: This study was funded by Eli Lilly and Company. Mr Fenske and Drs Ding, Morrow, and Smith are employed by Eli Lilly and Company. Drs Manjelievskaia, Moynihan, and Silver are employed by Merative. Drs Manjelievskaia, Moynihan, and Silver were employed by IBM Watson Health at the time of study completion. IBM Watson Health received funding from Eli Lilly and Company to conduct this study.

Burden of illness in aromatic l‐amino acid decarboxylase deficiency

AbstractObjectivesDeficiency of aromatic l‐amino acid decarboxylase (AADC), a key enzyme in monoamine synthesis, is an ultrarare disorder of monoamine synthesis estimated to affect fewer than 50 patients in the United States, although rates of misdiagnosis may be high. In its severe form, this congenital hypodopaminergic state results in profound hypotonia, developmental impairment, oculogyric crises, dystonia, dysautonomia, hypoglycemia, and premature death. The present study assesses the burden of illness for patients and caregivers by assessment of medical resource utilization (MRU) and quality of life (QOL) measures to enable future health technology assessment activities for emerging therapeutic options.MethodsData from patients’ medical records from the prior two years were examined to estimate burden in terms of direct costs; indirect care burden (including caregiver impact on sleep, health, and vocational opportunities) and QOL were also assessed using the BURQOL‐RD, AQoL‐6, and Visual Analogue Scale questionnaires.ResultsData were available for five US cases (three males, two females; age, 2–24 years, median, 5 years). MRU ranged from $6162.73 to $49 043.94 annually per patient. Although the proportion of costs (inpatient and outpatient visits, labs/tests, drugs, devices/durable medical equipment, and speech/physical therapy) varied greatly, the average annual direct cost of healthcare was approximately $25 000. Caregivers described profound care burden and QOL effects, with an average indirect care burden cost assessment of nearly $43 000 yearly. The mean economic burden of AADC deficiency per year was estimated at $68 000.ConclusionMRU and QOL varied considerably by patient. This is attributable to the disease's variable phenotypic severity and differing patient needs. Frequent contact with the healthcare system, however, is a constant for patients with AADC deficiency and their caregivers. Quantifying MRU and QOL for rare genetic diseases is critical for assessing the value of emerging targeted therapies.

SA76 Burden of Illness in Patients with Alzheimer’s Disease: A Systematic Review of Incidence, Prevalence, Comorbidities, Disability and Mortality

SA76 Burden of Illness in Patients with Alzheimer’s Disease: A Systematic Review of Incidence, Prevalence, Comorbidities, Disability and Mortality

Burden of Illness in Patients with Higher-Risk Myelodysplastic Syndromes By Baseline Transfusion Status

Introduction: Higher-risk myelodysplastic syndromes (HR-MDS) are rare hematopoietic cancers. While hypomethylating agents (HMAs) are the standard of care, transfusions are important to manage symptoms of anemia and thrombocytopenia. This study aims to better understand the transfusion burden in these patients at baseline and how it is impacted post-HMA initiation, as well as the healthcare resource utilization (HRU) and costs amongst patients with HR-MDS post-HMA initiation, according to baseline transfusion status. Methods: A retrospective observational study using the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database. Patients aged ≥66 years, who initiated HMAs after being diagnosed with refractory anemia with excess blasts (a proxy for HR-MDS) between January 1, 2009 and December 31, 2017 were included. Patients were followed up until December 31, 2019 or date of death, whichever occurred earlier. Baseline transfusion status was defined during the 8-week period prior to HMA initiation. Transfusion naïve (TN) patients had no platelet (PLT) or red blood cell (RBC) transfusion episodes. Transfusion users (TU) had ≥1 PLT and/or RBC transfusion episodes. Baseline TU were stratified into PLT TU only, RBC TU only, and PLT+RBC TU. Transfusion independence (TI) was defined as no transfusion episode during an 8-week period. The conversion of baseline TU to TI was evaluated by corresponding transfusion type on a weekly rolling basis post-HMA initiation for 6 months (i.e. week 0-7, week 1-8, through to week 18-25). All-cause HRU and costs (adjusted to 2021 US dollars) post-HMA initiation were evaluated in terms of per-patient-per-month (PPPM) by baseline transfusion status. Results: In total, 603 patients treated with HMAs met the study criteria (Figure 1). Overall, 42% (254/603) of patients were baseline TN vs 58% (349/603) who were baseline TU (median age: 76 vs 77 years; male: 37% vs 38%; Charlson Comorbidity Index ≥3: 50% vs 64%; median follow-up: 17.0 vs 10.4 months, respectively). Amongst baseline TU, 80% (278/349) were RBC TU only, 16% (56/349) were PLT+RBC TU, and 4% (15/349) were PLT TU only. The median treatment duration of baseline TU receiving HMAs was 6.2 months. By the end of the first 8 weeks post-HMA initiation (week 0-7), 15% of the baseline RBC TU converted to RBC TI, and <20% of baseline PLT+RBC TU converted to PLT+RBC TI. The proportion of baseline RBC TU only who converted to RBC TI post-HMA initiation increased steadily over time; while varying degrees of conversion of baseline PLT+RBC TU to PLT+RBC TI was observed. The conversion of baseline TU to corresponding type of TI stabilized by 17-24 weeks post-HMA initiation: 45% for baseline RBC TU only, and 41% for baseline PLT+RBC TU (Figure 2). Of the baseline PLT TU only cohort, fewer than 11 patients converted to PLT TI at any weekly rolling basis post-HMA initiation. Baseline TN was associated with a lower all-cause HRU PPPM relative to baseline TU: mean (standard deviation [SD]), respectively, was 0.3 [0.5] vs 0.8 [1.4] for inpatient episodes, 0.3 [0.4] vs 0.8 [1.6] for emergency room visits, and 3.3 [2.8] vs 5.9 [15.5] for outpatient visits, p<0.001. Similarly, baseline TN status was associated with a lower total cost of care PPPM compared with baseline TU (mean [SD] of $20,713 [$14,361] vs $36,159 [$99,174], p=0.007). Inpatient cost was the main contributor to the total cost of care (mean [SD] of $7,418 [$9,790] for baseline TN vs $10,722 [$21,823] for baseline TU, p=0.15). Conclusions: Findings from this study demonstrated the transfusion burden amongst patients with HR-MDS. Nearly 60% of patients with HR-MDS were baseline TU. Although the specific cause of HRU cannot be identified in the database, baseline transfusion use was associated with higher all-cause HRU and total cost of care relative to baseline TN status. The majority (80%) of the baseline TU were RBC TU only. While the conversion from baseline RBC TU only to RBC TI was low (15%) by the end of the first 8 weeks post-HMA initiation, it increased over time, and stabilized at 45% by week 17-24 post-HMA initiation. These findings emphasize that novel treatments are needed for more rapid conversion of baseline TU to TI, and more sustained TI. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

RWD137 A Real-World Study to Estimate the Burden of Illness for Patients with Diabetic Peripheral Neuropathic PAIN (DPNP)

Currently, there is no specific characterization of the burden of illness for (DPNP) in most Asian countries nor a dedicated standard of care. The purpose of this research is to understand the burden of illness of DPNP in China from a hospital system perspective.

A Retrospective Claims-Based Study Evaluating Clinical and Economic Burden Among Patients With Moderate to Severe Osteoarthritis Pain in the United States.

Background: There has been limited evaluation of medication adherence, healthcare resource utilization (HCRU), and healthcare costs over time in patients with osteoarthritis (OA), and stratification by pain severity level has not been reported. Assessing such longitudinal changes may be useful to patients and healthcare providers for tracking disease progression, informing treatment options, and employing strategies to optimize patient outcomes. Objectives: To characterize treatment patterns, HCRU, and costs over time in patients with moderate to severe (MTS) OA pain in the United States. Methods: We conducted a retrospective claims analysis, using IBM® MarketScan® databases, from 2013-2018. Eligible patients were aged ≥45 years with ≥12 months pre-index (baseline) and ≥24 months (follow-up) of continuous enrollment; index date was defined as a physician diagnosis of hip or knee OA. An algorithm was employed to identify MTS OA pain patients, who were propensity score matched with patients having non-MTS OA pain. Data were summarized using descriptive statistics and univariate analyses. Results: After propensity score matching, the overall OA pain cohorts consisted of 186 374 patients each: 61% were female, mean age was 63 years, and two-thirds (65.6%) were of working age (45-65 years). Sleep-related conditions, anxiety, and depression were significantly higher in the MTS OA pain cohort vs non-MTS (P<0.001). At baseline and 12- and 24-month follow-ups, receipt of prescription pain medications, HCRU, and direct medical costs were significantly higher in the MTS OA pain cohort (all P<0.01). Medication adherence was significantly higher in the MTS OA pain cohort for all medication classes except analgesics/antipyretics, which were significantly lower vs the non-MTS OA pain cohort (all P<0.0001). Conclusions: The burden of MTS OA pain is substantial, with patterns that show increasing medication use, HCRU, and costs vs non-MTS OA pain patients over time. Understanding the heterogeneity within the OA population may allow us to further appreciate the true burden of illness for patients in pain.

Prophylaxis with anti-activated factor XII for hereditary angioedema

Hereditary angioedema is a rare autosomal dominant genetic disorder characterised by recurrent, localised swellings in various tissues including the skin, genitals, abdomen, face, and oropharyngeal region. These attacks can be painful, debilitating, and life-threatening, substantially affecting quality of life. 1 Banerji A The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013; 111: 329-336 Google Scholar , 2 Bork K Hardt J Witzke G Fatal laryngeal attacks and mortality in hereditary angioedema due to C1-INH deficiency. J Allergy Clin Immunol. 2012; 130: 692-697 Google Scholar Inadequate control of the contact system, consisting of factor XII (FXII), plasma kallikrein, and high molecular weight kininogen, results in excessive bradykinin formation, which increases vascular permeability and thus causes angioedema attacks. 3 Fijen LM Bork K Cohn DM Current and prospective targets of pharmacologic treatment of hereditary angioedema types 1 and 2. Clin Rev Allergy Immunol. 2021; 61: 66-76 Google Scholar Prophylactic use of an anti-activated factor XII monoclonal antibody, garadacimab, for patients with C1-esterase inhibitor-deficient hereditary angioedema: a randomised, double-blind, placebo-controlled, phase 2 trialGaradacimab 200 mg and 600 mg every 4 weeks significantly reduced the number of monthly attacks versus placebo and was well tolerated during the study. Garadacimab is an efficacious, subcutaneous prophylaxis in patients with HAE-C1-INH and warrants phase 3 evaluation. Full-Text PDF

P585 Examining faecal incontinence and its impact on health-related quality of life in patients with Crohn’s perianal fistulas: results from a multi-country burden of illness study

Abstract Background Perianal fistulas (PAF) are a common complication associated with Crohn’s disease (CD), and patients with Crohn’s perianal fistulas (CPF) are more likely to experience faecal incontinence (FI) than patients with CD without PAF (non-PAF CD). A global study was conducted to assess the burden of illness in patients with CPF compared with patients with non-PAF CD. Here, we present data on the level of FI and its impact on quality of life (QoL). Methods This cross-sectional study was conducted in seven countries (France, Germany, Spain, UK, Canada, Australia and Japan) in patients aged ≥21 and ≤90 years with self-reported physician-diagnosed CD. Patients were classified as having non-PAF CD (cohort 1) or CPF without PAF-related surgery (cohort 2) or CPF with PAF-related surgery (cohort 3). Validated, general and disease-specific patient-reported outcome data were collected using a 45-min IRB/EC-approved web-enabled questionnaire. The level of FI was evaluated using the Revised Faecal Incontinence Score (RFIS) (1–20 scale, 0 = no incontinence, 4-week recall period) and the impact of FI on QoL in patients who had experienced FI using the Fecal Incontinence Quality of Life (FIQL) (1–5 scale, 1 = lower QoL). Data were analysed using descriptive statistics. Results Of the 929 patients recruited (cohort 1, n = 620; cohort 2, n = 174; cohort 3, n = 135), 58–69% were male and 55–67% were aged 21–40 years across all cohorts. A significantly higher proportion of patients in cohort 3 than cohort 1 had severe Crohn’s disease as classified by a physician (18% vs 9%, respectively; p &amp;lt; 0.05). Overall, 47% of patients reported experiencing FI and completed both the RFIS and FIQL. Significantly higher proportions of patients in cohorts 2 and 3 had experienced FI than in cohort 1 (59% and 59% vs 40%, respectively; p &amp;lt; 0.05), and a significantly higher proportion were currently experiencing FI in cohort 3 than in cohorts 1 and 2 (69% vs 37% and 45%, respectively; p &amp;lt; 0.05). Mean RFIS scores were significantly higher (worse) in cohorts 2 and 3 than cohort 1 (9.5 [p &amp;lt; 0.05] and 10.6 [p &amp;lt; 0.001] vs 8.2, respectively). Patients in cohort 2 had significantly lower (worse) FIQL scores than those in cohort 1 across three FIQL domains (lifestyle [2.2 vs 2.4], depression/self-perception [2.1 vs 2.3], embarrassment [2.2 vs 2.4]; p &amp;lt; 0.05), whereas patients in cohort 3 only reported significantly lower FIQL scores compared with cohort 1 in the embarrassment domain (2.2 vs 2.4; p &amp;lt; 0.05). Conclusion In this study almost half of the patients experienced faecal incontinence. Among those who experienced faecal incontinence, patients with PAF experienced a significantly higher impact on their quality of life compared with patients without PAF.

P516 Discrete choice experiment to examine patient preferences for surgical interventions in patients with Crohn’s perianal fistulas: results from a global burden of illness study

Abstract Background Perianal fistulas (PAF) are a common complication of Crohn’s disease (CD) and patients with Crohn’s perianal fistulas (CPF) experience low health-related quality of life and a higher burden of symptoms than patients with CD without PAF (non-PAF CD). Treatment options for PAF, including medical and surgical interventions, are associated with high rates of relapse or failure. This global study assessed the burden of illness in patients with CPF compared with patients with non-PAF CD. Here, we present data on patient satisfaction with currently available PAF treatments and the attributes that drive treatment choice for patients with CPF. Methods This cross-sectional study was conducted in seven countries (France, Germany, Spain, UK, Canada, Australia and Japan) in patients aged ≥21 and ≤90 years with self-reported physician-diagnosed CD. Patients were classified as non-PAF CD (cohort 1) or CPF without PAF-related surgery (cohort 2) or CPF with PAF-related surgery (cohort 3). Using a web-enabled questionnaire, patient satisfaction with current PAF treatment options was recorded using a 1–9 scale and relative treatment attribute preferences were assessed via a discrete choice experiment (DCE) that included six attributes (Figure 1). Patients evaluated two treatment profiles at a time (“choice tasks”) to identify their most preferred treatment of the choices available. Data were analysed using descriptive statistics. Results A total of 929 patients were recruited, 620 with non-PAF CD and 309 with CPF. Of those with CPF (cohort 2, n=174; cohort 3, n=135), 68% were male, 65% were aged 21–40 years and 83% were currently taking CD-related medication. The median number of PAF was the same in cohorts 2 and 3 (both 2.0), although cohort 3 had a higher proportion of patients with active PAF (119/135 [88%]) compared with cohort 2 (136/174 [78%]; p =0.022). In cohort 3, 105/135 (78%) patients had ≥3 PAF-related procedures/surgeries, and ≥1 and &amp;gt;3 post-operative complications were experienced by 117/135 (87%) and 34/135 (25%) patients, respectively. Mean satisfaction scores were moderate for the majority of PAF treatment options and similar in both cohorts (range 6.2–6.9), although cohort 3 had significantly less satisfaction with long-term seton placement than cohort 2 (6.2 vs 6.7, respectively; p =0.035). Among the tested attributes, post-operative discomfort and fistula healing rate were the most important attributes influencing treatment choice (Figure 1). Conclusion In this study, patients with CPF had moderate satisfaction with PAF treatment options, regardless of surgical intervention. The DCE demonstrated that patients preferred interventions with better healing rates and lower post-operative discomfort compared to other attributes tested.