Susan D. Mathias
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View article: Validation of the sunlight exposure diary and the erythropoietic protoporphyria impact questionnaire (EPIQ)
Validation of the sunlight exposure diary and the erythropoietic protoporphyria impact questionnaire (EPIQ) Open
Background Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare disorders that can negatively affect one’s health-related quality of life (HRQoL) because of pain from phototoxic reactions and the avoidance of sun …
View article: Self‐Reported Liver Disease and the Burden of Erythropoietic Protoporphyria
Self‐Reported Liver Disease and the Burden of Erythropoietic Protoporphyria Open
Erythropoietic protoporphyria (EPP) and X‐linked protoporphyria are metabolic disorders that cause skin phototoxicity and potential liver damage. We compared symptoms and impacts of phototoxic reactions, health‐related quality of life, and…
View article: Qualitative Research in Children and Parents of Children with Achondroplasia to Evaluate the Content Validity of Multiple Clinical Outcome Assessments
Qualitative Research in Children and Parents of Children with Achondroplasia to Evaluate the Content Validity of Multiple Clinical Outcome Assessments Open
Background. Achondroplasia is one of the most common skeletal dysplasias, characterized by disproportionate short stature and delayed motor development. Additional potential complications include bowed legs and hypotonia, otitis media, sle…
View article: Fatigue is distinct from sleepiness and negatively impacts individuals living with obstructive sleep apnea (OSA): results from qualitative research of individuals with OSA
Fatigue is distinct from sleepiness and negatively impacts individuals living with obstructive sleep apnea (OSA): results from qualitative research of individuals with OSA Open
Background We sought to identify important issues regarding symptoms and impacts of obstructive sleep apnea (OSA), to explore fatigue and sleepiness, and evaluate the content, clarity, and relevance of specific patient reported outcome (PR…
View article: Best Practices to Overcoming Challenges in Initiating Venetoclax for Patients with Chronic Lymphocytic Leukemia: Results from a United States Community-Based Healthcare Practitioner Survey
Best Practices to Overcoming Challenges in Initiating Venetoclax for Patients with Chronic Lymphocytic Leukemia: Results from a United States Community-Based Healthcare Practitioner Survey Open
Background: Initial treatment (tx) for patients (pts) with chronic lymphocytic leukemia (CLL) currently consists of either Bruton's tyrosine kinase inhibitor (BTKi)-based or venetoclax (Ven)-based combination regimens. While both are effec…
View article: The Development and Validation of the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH) 2.0
The Development and Validation of the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH) 2.0 Open
Background The Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) was initially developed in 2018 and includes three versions: pediatric, adult, and caregiver. The original versions are lengthy, and scoring can be complex an…
View article: Qualitative and Psychometric Evaluation of 29-Item Patient-Reported Outcomes Measurement Information System® to Assess General Health-Related Quality of Life in Patients With Moderately to Severely Active Inflammatory Bowel Disease
Qualitative and Psychometric Evaluation of 29-Item Patient-Reported Outcomes Measurement Information System® to Assess General Health-Related Quality of Life in Patients With Moderately to Severely Active Inflammatory Bowel Disease Open
PROMIS-29 is valid, reliable, and responsive for assessing general health-related quality of life and treatment response in IBD clinical trials.
View article: Development and Content Validation of Novel Patient-Reported Outcome Measures to Assess Disease Severity and Change in Patients with Erythropoietic Protoporphyria: The EPP Impact Questionnaire (EPIQ)
Development and Content Validation of Novel Patient-Reported Outcome Measures to Assess Disease Severity and Change in Patients with Erythropoietic Protoporphyria: The EPP Impact Questionnaire (EPIQ) Open
PRO measures are needed to assess important aspects of HRQoL and evaluate therapeutic response. These PRO measures are unique in assessing overall severity and change in EPP.
View article: Qualitative and psychometric evaluation of the PROMIS®-Fatigue SF-7a scale to assess fatigue in patients with moderately to severely active inflammatory bowel disease
Qualitative and psychometric evaluation of the PROMIS®-Fatigue SF-7a scale to assess fatigue in patients with moderately to severely active inflammatory bowel disease Open
Background This study evaluated the content validity and psychometric properties of the Patient-Reported Outcomes Measurement Information System® (PROMIS)-Fatigue Short Form 7a (SF-7a) v1.0 scale to determine its suitability in clinical tr…
View article: SAT622 Matched Physician- And Patient-reported Responses Of Acromegaly Symptom Control: Comparisons From The Macro Registry
SAT622 Matched Physician- And Patient-reported Responses Of Acromegaly Symptom Control: Comparisons From The Macro Registry Open
Disclosure: E.B. Geer: Consulting Fee; Self; Amryt. Research Investigator; Self; Ionis, Amryt. W. Huang: Consulting Fee; Self; Amryt. Speaker; Self; Amryt. M.B. Gordon: Consulting Fee; Self; Recordati, Novo Nordisk. Research Investigator; …
View article: PSAT103 Qualitative Research in Children with Achondroplasia and Parents of Children with Achondroplasia: Medical Challenges and Impacts
PSAT103 Qualitative Research in Children with Achondroplasia and Parents of Children with Achondroplasia: Medical Challenges and Impacts Open
Background Achondroplasia (ACH) is the most common form of non-lethal skeletal dysplasia affecting 1 in 25,000 live births. Individuals with ACH experience various medical symptoms/complications and impacts during their lifetime. Qualitati…
View article: PSAT102 Qualitative Research in Children and Parents of Children with Achondroplasia to Cognitively Debrief Three Patient-Reported Outcome Measures and Confirm the Content Validity of a Clinical-Assessed Measure
PSAT102 Qualitative Research in Children and Parents of Children with Achondroplasia to Cognitively Debrief Three Patient-Reported Outcome Measures and Confirm the Content Validity of a Clinical-Assessed Measure Open
Background Achondroplasia (ACH) is the most common non-lethal form of skeletal dysplasia affecting 1 in 25,000 live births. Individuals with ACH experience various medical, emotional, and functional challenges during their lifetime. Qualit…
View article: Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the <scp>TouchStone H</scp>ealthcare <scp>P</scp>rovider <scp>S</scp>urvey
Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the <span>TouchStone H</span>ealthcare <span>P</span>rovider <span>S</span>urvey Open
Background Systemic mastocytosis (SM) is a rare clonal neoplasm driven by the KIT D816V mutation and has a broad range of debilitating symptoms. In this study, the authors evaluated SM disease perceptions and management strategies among US…
View article: Patient‐reported outcomes among patients with systemic mastocytosis in routine clinical practice: Results of the <scp>TouchStone SM P</scp>atient <scp>S</scp>urvey
Patient‐reported outcomes among patients with systemic mastocytosis in routine clinical practice: Results of the <span>TouchStone SM P</span>atient <span>S</span>urvey Open
Background Systemic mastocytosis (SM) is a rare clonal neoplasm driven by KIT D816V and other mutations. Data were collected from the patient perspective on disease burden and included an SM‐specific symptom assessment tool. Methods US adu…
View article: Disease and Treatment-Related Burden in Patients With Acromegaly Who Are Biochemically Controlled on Injectable Somatostatin Receptor Ligands
Disease and Treatment-Related Burden in Patients With Acromegaly Who Are Biochemically Controlled on Injectable Somatostatin Receptor Ligands Open
Medical treatment for acromegaly commonly involves receiving intramuscular or deep subcutaneous injections of somatostatin receptor ligands (SRLs) in most patients. In addition to side effects of treatment, acromegaly patients often still …
View article: Determination of thresholds for minimally important difference and clinically important response on the functional outcomes of sleep questionnaire short version in adults with narcolepsy or obstructive sleep apnea
Determination of thresholds for minimally important difference and clinically important response on the functional outcomes of sleep questionnaire short version in adults with narcolepsy or obstructive sleep apnea Open
Purpose This study estimated thresholds for clinically important responses and minimally important differences for two indicators of improvement for the 10-item version of the functional outcomes of sleep questionnaire (FOSQ-10). Methods P…
View article: <scp>I‐WISh</scp>: A wish list for immune thrombocytopenia quality of life indicators becomes reality
<span>I‐WISh</span>: A wish list for immune thrombocytopenia quality of life indicators becomes reality Open
In this issue of the American Journal of Hematology, two articles by Nichola Cooper and colleagues describe results from the immune thrombocytopenia (ITP) World Impact Survey (I-WISh) and the ITP Life Quality Index (ILQI) among 1507 adults…
View article: Relationship between sleep efficacy endpoints and measures of functional status and health‐related quality of life in participants with narcolepsy or obstructive sleep apnea treated for excessive daytime sleepiness
Relationship between sleep efficacy endpoints and measures of functional status and health‐related quality of life in participants with narcolepsy or obstructive sleep apnea treated for excessive daytime sleepiness Open
This study examined the correlation between improvements in excessive daytime sleepiness in participants with obstructive sleep apnea or narcolepsy and changes in functional status, work productivity and health‐related quality of life. Dat…
View article: An evaluation of the Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) in adult patients with acromegaly, including correlations with other patient-reported outcome measures: data from two large multicenter international studies
An evaluation of the Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) in adult patients with acromegaly, including correlations with other patient-reported outcome measures: data from two large multicenter international studies Open
Purpose The Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ) is a new patient-reported outcome (PRO) measure for patients with acromegaly receiving injectable somatostatin analogs (SSAs) to assess clinical symptoms and adverse dr…