Percorrer por autor "Malaty, Irene A."
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- Burden and disutility of sleep disturbance and early morning OFF symptoms in people with advancing Parkinson’s disease : a vignette-based approach using the EQ-5D-5LPublication . Domingos, Josefa; Arija, Pablo; Malaty, Irene A.; Pahwa, Rajesh; Chaudhuri, K. Ray; Boeri, Marco; Kuharic, Maja; Lalla, Anjana; Baldwin, Zachary; Yan, Connie H.; Heisen, Marieke; Mohan, Divya; Penton, HannahBackground: Parkinson’s disease (PD) is characterized by motor and non-motor symptoms that fluctuate as oral medication wears off, causing periods when symptoms return (“OFF” time) and periods of control (“ON” time). These fluctuations often occur at night and early morning. Sleep disturbance (SD) and early-morning OFF time (EMO) have been shown to decrease health-related quality of life (HRQoL) in people with PD (PwP). However, limited evidence exists on how these symptoms impact health state utility values (HSUVs) used in economic modeling. This study aimed to estimate the burden and disutility of SD and EMO in PwP through vignettes valued using the EQ-5D-5 L. Methodology: An online survey was completed by adults (≥ 30 years) with self-reported PD diagnosis for ≥ 5 years and ≥ 2 h/day of OFF time, on oral PD medications, and residing in the US or UK. The survey included EQ-5D-5 L and EQ-VAS assessments for 4 vignettes—No SD or EMO, SD (without EMO), EMO (without SD), and Both SD and EMO—plus questions exploring the burden of SD and EMO. EQ-5D-5 L responses were converted into US utility values to estimate HSUVs and the disutility of SD and EMO. Results: Seventy-five individuals completed the survey; 52.00% were male, and 78.66% resided in the US. The average age was 64.32 years, with a mean time since diagnosis of 9.82 years and an average OFF time of 3.91 h/day. In the previous week, 96.00% and 98.67% of respondents reported experiencing SD and EMO, respectively. The presence of either SD (HSUV: 0.796) or EMO (HSUV: 0.701) alone resulted in lower EQ-5D-5 L utilities, with the presence of both valued least (HSUV: 0.528), compared with when both SD and EMO were absent (HSUV: 0.911). EQ-VAS scores ranged from 81.01 for no symptoms to 48.41 for both. Conclusions: SD and EMO impose a significant burden on PwP and decrease HSUVs. As SD and EMO were associated with significant disutility, they should be incorporated into economic models to comprehensively assess PD treatment benefits. Raising awareness among PwP and healthcare providers and prioritizing treatments that minimize SD and EMO can reduce their burden and improve HRQoL for PwP.
- Does the 5–2-1 criteria identify patients with advanced Parkinson's disease? Real-world screening accuracy and burden of 5–2-1-positive patients in 7 countriesPublication . Malaty, Irene A.; Martinez-Martin, Pablo; Chaudhuri, K. Ray; Odin, Per; Skorvanek, Matej; Jimenez-Shahed, Joohi; Soileau, Michael J.; Lindvall, Susanna; Domingos, Josefa; Jones, Sarah; Alobaidi, Ali; Jalundhwala, Yash J.; Kandukuri, Prasanna L.; Onuk, Koray; Bergmann, Lars; Femia, Samira; Lee, Michelle Y.; Wright, Jack; Antonini, AngeloBackground: The burden of Parkinson’s disease (PD) worsens with disease progression. However, the lack of objective and uniform disease classification challenges our understanding of the incremental burden in patients with advanced Parkinson’s disease (APD) and suboptimal medication control. The 5–2-1 criteria was proposed by clinical consensus to identify patients with advancing PD. Our objective was to evaluate the screening accuracy and incremental clinical burden, healthcare resource utilization (HCRU), and humanistic burden in PD patients meeting the 5–2-1 screening criteria. Methods: Data were drawn from the Adelphi Parkinson’s Disease Specific Program (DSP™), a multi-country point-in-time survey (2017–2020). People with PD who were naive to device-aided therapy and on oral PD therapy were included. Patients meeting the 5–2-1 screening criteria had one or more of the three clinical indicators of APD: (i) ≥5 doses of oral levodopa/day, OR (ii) “off” symptoms for ≥2 h of waking day, OR (iii) ≥1 h of troublesome dyskinesia. Clinician assessment of PD stage was used as the reference in this study. Clinical screening accuracy of the 5–2-1 criteria was assessed using area under the curve and multivariable logistic regression models. Incremental clinical, HCRU, and humanistic burden were assessed by known-group comparisons between 5 and 2-1-positive and negative patients. Results: From the analytic sample (n = 4714), 33% of patients met the 5–2-1 screening criteria. Among physician-classified APD patients, 78.6% were 5–2-1 positive. Concordance between clinician judgment and 5–2-1 screening criteria was > 75%. 5–2-1-positive patients were nearly 7-times more likely to be classified as APD by physician judgment. Compared with the 5–2-1-negative group, 5–2-1-positive patients had significantly higher clinical, HCRU, and humanistic burden across all measures. In particular, 5–2-1-positive patients had 3.8-times more falls, 3.6-times higher annual hospitalization rate, and 3.4-times greater dissatisfaction with PD treatment. 5–2-1-positive patients also had significantly lower quality of life and worse caregiver burden. Conclusions: 5–2-1 criteria demonstrated potential as a screening tool for identifying people with APD with considerable clinical, humanistic, and HCRU burden. The 5–2-1 screening criteria is an objective and reliable tool that may aid the timely identification and treatment optimization of patients inadequately controlled on oral PD medications.
- Exploring preferences and priorities in advanced Parkinson’s disease : a discrete choice experimentPublication . Arija, Pablo; Domingos, Josefa; Malaty, Irene A.; Pahwa, Rajesh; Chaudhuri, K. Ray; Antonini, Angelo; Penton, Hannah; Heisen, Marieke; Yan, Connie H.; Kukreja, Pavnit; Shirneshan, Elaheh; Lee, Inyoung; Shah, Megha; Parra, Juan Carlos; Boeri, MarcoIntroduction: Treatments for advanced Parkinson’s disease (aPD) are differentiated by efficacy, safety, and modality-related characteristics. As the disease progresses and motor fluctuations worsen, many patients require more frequent dosing or consideration of device-aided therapies, including subcutaneous infusions, intestinal gel delivery systems, or deep brain stimulation. Assessing treatment preferences is valuable to ensure people with aPD (PwP) and care partners (CPs) are satisfied with a treatment’s impact on both motor function and quality of life, potentially increasing adherence and effectiveness. Methods: A total of 304 participants (223 PwP, 81 CPs) from the USA, UK, and Germany were included in the study. A discrete choice experiment (DCE) was used to elicit preferences over treatment characteristics. In the DCE, respondents were presented with a series of choice tasks, each consisting of two hypothetical treatments described by varying levels of seven attributes: daily hours of ON time without troublesome dyskinesia (ONwoTD), frequency of early morning OFF time (EMO), risk of mild-to-moderate skin reactions, risk of severe side effects requiring hospitalization, route of administration (ROA), frequency of pill regimen, and frequency of device maintenance. Analyses with a random parameter logit model were used to estimate attribute conditional relative importance (CRI) and explore how people would trade off across attributes. Results: The average PwP age was 65.7 years (SD 8.6), time since diagnosis was 10.0 years (SD 4.4), and self-reported OFF time was 4.0 h/day (SD 2.4). Within the survey design, ROA emerged as the most important attribute (CRI 35.3), followed by hours of ONwoTD (CRI 26.4). All other attributes were of similar importance. Nonsurgical treatments were strongly preferred, with oral pills being the most preferred, followed by infusion device without surgery (subcutaneous infusion). Conclusions: PwP prioritized efficacy (ONwoTD) and ROA when considering treatment options. Understanding these preferences may enhance informed and meaningful decision-making between healthcare providers and PwP.