Publications by authors named "Roger E Lamoureux"
Background: The Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) (©Blueprint Medicines Corporation), a 12-item daily diary that assesses 11 signs and symptoms of indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM), was psychometrically evaluated among patients with ISM. Additionally, thresholds of the ISM-SAF total symptom score (TSS) to distinguish patients with moderate to severe symptoms from those with mild symptoms were evaluated.
Methods: The ISM-SAF was completed daily as an electronic diary in a prospective, observational study utilizing an online survey of patients with ISM in the United States.
Background: Daily injections of recombinant human growth hormone are the standard of care to treat growth failure due to pediatric growth hormone deficiency (GHD). While effective, daily injections are burdensome and can compromise adherence. In recent years, novel injection treatments requiring less frequent administration for growth hormone deficiency (GHD) have been developed.
View Article and Find Full Text PDFArticle Synopsis
- The study focused on developing two questionnaires to assess the signs and symptoms of eosinophilic esophagitis (EoE) in children ages 1 to less than 12 years, taking into account that younger patients may struggle to self-report their symptoms.
- Researchers conducted literature reviews, expert meetings, and interviews with children and their caregivers to identify relevant symptoms and ensure the questionnaires were understandable and comprehensive.
- The result was the Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire for self-reporting in older children (PESQ-P) and a caregiver-reported version (PESQ-C), which both showed strong content validity for use in clinical trials.
Background: Advanced systemic mastocytosis (AdvSM), indolent systemic mastocytosis (ISM), and smoldering systemic mastocytosis (SSM) are rare diseases characterized by neoplastic mast cell infiltration of more than one organ. A content-valid patient-reported outcome (PRO) questionnaire that assesses relevant signs and symptoms that are important and understandable to individuals with a condition is critical for assessing new treatment benefit as well as supporting product labeling claims. Notably, no such PRO questionnaire has been developed in accordance with regulatory and scientific guidelines for use in AdvSM, ISM, and SSM patient populations.
View Article and Find Full Text PDFPurpose: Fabry disease is a rare multisystemic disorder caused by functional deficiency of the lysosomal enzyme alpha-galactosidase A. Gastrointestinal (GI) signs and symptoms are among the earliest clinical manifestations in patients with Fabry disease but are often nonspecific, misdiagnosed, and untreated. No instruments have been developed specifically to assess GI signs and symptoms in Fabry disease.
View Article and Find Full Text PDFArticle Synopsis
- Current r-hGH replacement therapy for growth hormone deficiency (GHD) involves daily subcutaneous injections, which are burdensome for patients and may lead to poor adherence to treatment.
- To improve treatment compliance and outcomes, qualitative research was conducted to develop the Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD) that captures patient experiences related to these injections.
- The LIQ-GHD was tested through interviews and a cross-sectional study to assess its content, reliability, and validity across different patient age groups, using expert insights and literature review to refine the questionnaire.
Article Synopsis
- - The study aims to provide evidence-based recommendations for determining sample size in qualitative concept elicitation (CE) interviews, focusing on the point of saturation where no new data is expected.
- - A retrospective evaluation of 26 CE interview studies revealed that most symptom concepts saturated around the 10th to 25th interviews, with 99% of concepts emerging by the 25th interview.
- - The findings offer practical guidance for researchers on how many interviews might be necessary for saturation in qualitative studies related to clinical outcome assessment (COA) instrument development, addressing a key gap in qualitative research.
Introduction: Patient-reported outcome (PRO) measures serve to capture vital patient information not otherwise obtained by primary study endpoints. This paper examines how PROs are utilized as endpoints in industry-sponsored metastatic breast cancer clinical trials.
Methods: A search was conducted in the clinicaltrials.