Leighann Litcher-Kelly

• Managing Director at Adelphi Values

Background CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is a newly identified condition with an estimated worldwide prevalence of < 100 patients. Patient interviews can ensure that what is important to patients is assessed in a clinical trial program. Due to the rare an...

Background CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is ultra-rare (< 100 children or young adults worldwide) and potentially fatal. The study used mixed-methods approaches to assess how pozelimab impacts the signs and symptoms of CHAPLE disease from the patient pers...

Individuals living with Crohn’s disease (CD) experience burdensome symptoms. As such, it is important to measure CD symptom severity in clinical research. The goal of this study was to evaluate the content validity, psychometric performance, and score interpretability of a new patient-reported instrument, the Crohn’s Symptom Severity (CSS) question...

Objectives Bowel urgency is an impactful core symptom of ulcerative colitis (UC). Patient-reported outcome (PRO) questionnaires have been developed and used to assess the patient experience of this important symptom. The objective of this paper is to present evidence from qualitative research conducted to support the use and interpretation of selec...

Background: Atopic dermatitis (AD) is a chronic inflammatory skin disease characterized by pruritus, skin pain, and sleep impacts, which are only reportable by patients themselves. The goal of this research is to evaluate the reliability, validity, and interpretability of the scores from three patient-reported outcome measures within the context o...

Background Patients with Crohn’s disease (CD) or ulcerative colitis (UC) frequently experience fatigue, although it is often overlooked in medical research and practice.AimsTo explore patients’ experience of fatigue and evaluate content validity, psychometric properties, and score interpretability of the Functional Assessment of Chronic Illness The...

IntroductionThree patient-reported outcome (PRO) questionnaires—Worst Pruritus Numerical Rating Scale (WP-NRS), Atopic Dermatitis Symptom Scale (ADerm-SS), and Atopic Dermatitis Impact Scale (ADerm-IS)—were developed to assess the symptoms and impacts of atopic dermatitis (AD). Severity strata for these PROs are needed to aid in their interpretatio...

Background Recurrent pericarditis is characterized by painful flares and inflammation, which negatively impact health‐related quality of life. RHAPSODY (rilonacept inhibition of interleukin‐1 alpha and beta for recurrent pericarditis: a pivotal symptomatology and outcomes study) evaluated the efficacy and safety of rilonacept (IL‐1α and ‐β cytokine...

Background Pediatric patients with eosinophilic esophagitis (EoE) experience heterogenous symptoms and patients’ age may preclude reliable self-report of symptoms. Objective The goal of this study was to develop a patient-reported outcome (PRO) and an observer-reported outcome (ObsRO) questionnaire to evaluate the signs and symptoms of EoE in pedi...

Background Impact of recurrent pericarditis (RP) on patient health-related quality of life (HRQoL) was evaluated through qualitative patient interviews and as an exploratory endpoint in a Phase 2 trial evaluating the efficacy and safety of rilonacept (IL-1α/IL-1β cytokine trap) to treat RP. Methods Qualitative interviews were conducted with ten ad...

Background: Impact of recurrent pericarditis (RP) on patient health-related quality of life (HRQoL) was evaluated through qualitative patient interviews and as an exploratory endpoint in a Phase 2 trial evaluating the efficacy and safety of rilonacept (IL-1α/IL-1β blocker) to treat RP. Methods: Qualitative interviews were conducted with ten adults...

Background: Recurrent pericarditis (RP) episodes and conventional treatments result in morbidity, but the impact on patient health-related quality of life (HRQoL) has not been quantified. The Phase 2 trial NCT03980522 of rilonacept (IL-1α/IL-1β inhibitor) evaluated HRQoL. Methods: Patients with active RP who were symptomatic at Baseline (A-RP, n=16...

Please indicate where the abstract has been published before: Blood 2018 Background Sickle cell disease (SCD), an inherited blood disorder, impacts multiple organs leading to variable clinical presentation. In addition to severe pain episodes, patients may experience chronic symptoms (eg, pain, tiredness). Assessing treatment benefit using a patie...

Mucopolysaccharidosis I (MPS-I) is a rare autosomal recessive genetic disorder that can have physical, social, and emotional impacts. The MPS Health Assessment Questionnaire (MPS-HAQ) was developed to assess these impacts and includes 52 items across 10 domains of activities of daily living (i.e., eating/drinking, dressing, bathing, grooming, tooth...

Objectives: Atopic dermatitis (AD) is a chronic, relapsing skin condition, with signs and symptoms that impact patients’ lives and are best measured from the patient perspective. Therefore, there is a need for AD-specific questionnaires that are consistent with Food and Drug Administration guidance and best measurement practices, assessing sign and...

OBJECTIVES: Sickle cell disease (SCD), a group of inherited blood disorders, can impact multiple organs and can lead to variable clinical presentations. In addition to severe pain crises, patients may experience chronic symptoms (e.g. pain, tiredness). Assessing treatment benefit using a patient-reported outcome (PRO) measurement strategy can be cr...

Objective Evidence-based recommendations for the a priori estimation of sample size are needed for qualitative concept elicitation (CE) interview studies in clinical outcome assessment (COA) instrument development. Saturation is described as the point at which no new data is expected to emerge from the conduct of additional qualitative interviews....

Aims: Many treatment options are available for people with Type 2 Diabetes (T2D). While the goal of treatment is to reach target HbA1c levels, the psychological experience may be more tangible and meaningful for the patient. Together with biomarkers, Patient-Reported Outcome (PRO) data provide a holistic understanding of the clinical and psychosoc...

In order to assess potential associations between autism spectrum disorder (ASD) phenotype, functional GI disorders and fecal microbiota, we recruited simplex families, which had only a single ASD proband and neurotypical (NT) siblings, through the Simons Simplex Community at the Interactive Autism Network (SSC@IAN). Fecal samples and metadata rela...

Objectives: Research on the health of workers involved in the cleanup after the attack on the World Trade Center (WTC) on September 11, 2001, has documented high rates of psychological distress and upper gastrointestinal (GI) symptoms. The current article examines the concurrent and longitudinal associations of psychological distress with developm...

Unlabelled: There are many types of pain assessments available to researchers conducting clinical trials, ranging from simple, single-item Visual Analog Scale (VAS) questions through extensive, multidimensional inventories. The primary question addressed in this survey of top-tier medical journals was: Which pain assessments are most commonly used...

Self-report items from the Crohn's Disease Activity Index (CDAI) are completed using a 7-day paper diary; however, there are reports of protocol deviations by both patients and researchers/clinicians. The self-report literature has demonstrated these deviations can distort the information collected by introducing recall bias, especially for subject...

There are many complexities and difficulties inherent in reliably and validly measuring psychological and behavioral constructs. This chapter discusses a collection of techniques for capturing peoples' self-reports, including private, subjective states; behaviors emitted by an individual; and qualities of the environment. It addresses the measureme...

Unlabelled: The purpose of this article is to explore the multidimensionality of the pain experience for patients with chronic pain by using a within-person, longitudinal approach. An Ecological Momentary Assessment design with a patient electronic diary was used to collect random momentary pain assessments several times a day for 2 weeks. We exam...

Patient self-reports are the primary method for capturing the experience of pain, and diaries are often used to collect patient self-reports. This study was designed to determine if momentary monitoring of pain with an electronic diary affected pain levels over time, if it affected weekly recall of pain, and if daily sampling density affected compl...

Over the past two decades, our research group investigated the psychological well-being of mothers of young children in the aftermath of the nuclear power plant accident at Three Mile Island (TMI) in central Pennsylvania and the melt down at the Chernobyl nuclear power plant in northwest Ukraine. In spite of differences in culture and magnitude, th...

This paper examines the psychometric properties of the Children's Somatization Inventory (CSI) in 600 10-12-year old children in Kyiv, Ukraine, replicating and extending the original findings from a sample in Nashville, Tennessee (J. Garber et al. 1991). The Kyiv children had significantly lower CSI total scores and reported significantly fewer sym...

Relaxation techniques can lead to symptom reduction and improved pulmonary functioning in asthmatics, although the mechanism is not clear. One possibility is by influencing cortisol secretion, as cortisol is implicated in inflammatory processes and relaxation has been shown to alter cortisol secretion in healthy individuals. This study explored the...

This study used a parent-completed, DSM-IV-referenced rating scale to examine prevalence rates of attention-deficit/hyperactivity disorder (ADHD) behaviors and differences between subtypes in 10- to 12-year-old Ukrainian children. During 1997, a total of 600 parents and children residing in Kyiv, Ukraine, and their teachers participated in extensiv...

This paper examines the cognitive and neuropsychological functioning of children who were in utero to age 15 months at the time of the Chornobyl disaster and were evacuated to Kyiv from the 30-kilometer zone surrounding the plant. Specifically, we compared 300 evacuee children at ages 10–12 with 300 non-evacuee Kyiv classmates on objective and subj...