All posts by Mary Jo Strobel

Share Your EGPA Perspectives

APFED is working in partnership with a Medscape, the largest provider of continuing medical education to develop an accredited activity to educate physicians about eosinophilic granulomatosis with polyangiitis (EGPA), formerly known as Churg-Strauss Syndrome. This educational program will also provide the learners with perspective of those who live with EGPA (or their caregivers).

We are currently seeking five people with EGPA (or their caregiver) to participate in a telephone interview with Medscape to capture their perspective to include in this program.

The person being interviewed will receive an honorarium for taking part in the interview.

Interviews will be conducted as soon as possible — please responded by February 28th.

To learn more and to complete a short questionnaire to indicate your interest, please visit this page: https://apfed.org/egpa-patient-perspective/

 

Highlights from ACG 2019

This week, APFED was onsite at the American College of Gastroenterology’s 84th Annual Scientific Meeting and Postgraduate Course (ACG 2019) where attendees from around the world gathered to learn the latest scientific advances in gastrointestinal research and treatment of digestive diseases.

APFED had a table in the exhibit hall to offer clinicians professional resources to increase their understanding of the latest in diagnostics and treatments of these conditions, as well as educational material to share with their patients.


There were several exciting research posters presented throughout the meeting, including three in which APFED co-authored:

    • “Unmet Need for Additional Resources to Support Successful Transition of Care from Pediatric to Adult Providers for the Management of Eosinophilic Gastrointestinal Diseases,” presented by Gary Falk, MD, University of Pennsylvania
    • “Shared Decision Making in Eosinophilic Esophagitis Is Associated With Treatment Satisfaction but Is Inadequately Practiced,” presented by Joy W. Chang, MD, University of Michigan Medical Center
    • “Elucidation of Patient Motivators and Barriers to Pursuing Treatments for Eosinophilic Esophagitis,” presented by Joy W. Chang, MD, University of Michigan Medical Center

Other highlights from the meeting that are of interest to our community:

  • Evan Dellon, MD, from University of North Carolina at Chapel Hill, gave a lecture titled, “New Concepts in the Treatment of Eosinophilic” in which he reviewed updates in the epidemiology, nomenclature, diagnostic processes, and management of eosinophilic esophagitis (EoE).
  • Ikuo Hirano, MD, from Northwestern University in Chicago, presented data from the phase 3 trial of oral budesonide suspension (OBS) for the treatment of EoE. The study demonstrated its efficacy end points of symptom improvement and histologic efficacy.
    Read the press release from Takeda, the company investigating OBS for EoE.
    See below for a related interview with Dr. Hirano from MD Mag.
  • A session titled “Eosinophilic Esophagitis: Key Updates and Optimal Management” featured speakers Calies D. Menard-Katcher, MD (Children’s Hospital Colorado), who described the epidemiology and clinical presentation of pediatric EoE and discussed the transition of patients from pediatric to adult care; Nicholas J. Shaheen, MD (University of North Carolina at Chapel Hill), who discussed current diagnostic guidelines; and Kristle L. Lynch, MD (Perelman School of Medicine, University of Pennsylvania), who talked about the standard of care and emerging therapies.
  • Evan Dellon, MD, from University of North Carolina at Chapel Hill, presented a late-breaking abstract from a phase 2 trial of a potential therapy known as AK002 for eosinophilic gastrointestinal diseases. Allakos, the company developing this therapy is planning to initiate a Phase 3 study in EG and EGE and a Phase 2/3 study in EOE in 2020.
    See below for a related interview with Dr. Dellon from MD Mag.

The next annual meeting of ACG will take place Oct. 23-28 in Nashville, TN.

Highlights from the 2019 NASPGHAN/CPNP/APGNN Annual Meeting

The 2019 NASPGHAN/CPNP/APGNN Annual Meeting was held in Chicago Oct 16-19. APFED leadership attended the meeting to attend sessions and meet with providers, investigators, and other stakeholders to raise awareness, educate, and discuss the challenges and needs of patients with eosinophil-associated diseases and how those might be met.

We were also excited to once again offer abstract awards to outstanding research abstracts on eosinophil-associated gastrointestinal diseases (EGIDs). APFED funded two $750 awards to the best-scoring abstracts which are intended to help defray travel costs to the meeting so that the researchers may present their findings to their peers. Abstract award winners may go on to successfully compete for grants from APFED or from other funding mechanisms. Read about the abstract winners here.

Below, we summarize other highlights from the meeting that are of interest to our community:

  • The Association of Pediatric Gastroenterology and Nutrition Nurses (APGNN) offered sessions focused on pediatric feeding disorders. These talks were designed to teach attendees about primary domains that contribute to feeding disorders and the importance of early diagnosis and intervention, as well as how to support families of children with feeding disorders.
  • Drs. Amir Kagawalla (Northwestern University Feinberg School of Medicine) and Calies Menard-Katcher (Children’s Hospital Colorado) hosted a session that offered attendees the opportunity to discuss challenging cases in EoE.
  • Dr. Prerana Williamson from the University of California/Rady’s Children’s Hospital, San Diego presented research her team conducted about PAI-1 as an epithelial marker of disease severity in pediatric EoE.
  • Dr. Joshua Wechsler from the Ann & Robert H Lurie Children’s Hospital in Chicago spoke to attendees about PPI responsiveness in patients with EoE and new international consensus regarding the role of PPIs in diagnostics and treatment. A lay-friendly summary document is available from APFED.
  • The NASPGHAN Council for Pediatric Nutrition Professionals (CPNP) hosted a session specific to home parenteral nutrition and the journey from inpatient consultation to a successful transition home.
  • A session specific to upper GI interventions included talks included the types of endoscopic procedures for upper GI motility and functional disorders and the utility of these techniques in the pediatric population, including the esophageal string test, cytosponge, EndoFLIP and unsedated transnasal endoscopy. This discussion was led by Dr. Joel Friedlander of Children’s Hospital of Colorado. EndoFLIP is technology that measures the distensibility, or amount of “stretch” of the esophagus, which is believed has potential to be a marker of early fibrosis in EoE. Unsedated transnasal endoscopy which involves local anesthetic and passing endoscope into the esophagus via the nose. You may view a video of Dr. Friedlander discussing and demonstrating this technology online.
  • A talk designed to educate attendees about the barriers to getting medical foods covered by insurance company and what NASPGHAN is doing to help support efforts in this area (see our page about the Medical Nutrition Equity Act to learn more).
  • A number of poster abstracts that were presented onsite included case studies and breaking research. Poster titles and summaries may be found on the NASPGHAN website.

The 2020 annual NASPGHAN Meeting will be held Nov 04 – 09, 2020 in San Diego.

Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Receives Renewed Federal Funding

For Immediate Release

American Partnership for Eosinophilic Disorders
P.O. Box 29545
Atlanta, GA 30359
www.apfed.org

Contact: Mary Jo Strobel, Executive Director, (713) 493-7749, mjstrobel@apfed.org

 

Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Receives Renewed Federal Funding
APFED to continue participation in multi-center collaboration

(Atlanta, GA)— The American Partnership for Eosinophilic Disorders (APFED), a non-profit advocacy organization established in 2001, is pleased to share that the National Institutes of Health (NIH)  has awarded continued funding to the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) to research eosinophil-associated gastrointestinal diseases (EGIDs) and train investigators to conduct clinical research.

The five-year, $7.57 million grant (U54AI117804) is funded by the Office of Rare Diseases Research, which is part of the NIH’s National Center for Advancing Translational Sciences, as part of the Rare Diseases Clinical Research Network, and by the National Institute of Allergy and Infectious Diseases, the National Center for Advancing Translational Sciences, and the National Institute of Diabetes and Digestive and Kidney Diseases. Thanks to the generous donations of community members, APFED also provides supplemental funding to CEGIR through its HOPE on the Horizon Research Grant Program.

CEGIR brings together scientists, health care providers, patients, and professional organizations such as APFED to bring transformative changes to the care of patients with EGIDs. CEGIR first formed in 2014 after receiving $6.25 million through the NIH to research EGIDs, focusing its efforts on patients with eosinophilic esophagitis (EoE), eosinophilic gastritis (EG), and/or eosinophilic colitis (EC). During its first 5-year cycle of funding, CEGIR launched a long-term study and follow-up of patients with EoE, EG, and EC as well as several other studies to help better understand the natural history of these diseases and which treatments work for which diseases.

In addition to offering supplemental grant opportunities to CEGIR, APFED works in concert with CEGIR to ensure the patient perspective is included in all aspects of its work and to help recruit patients into critical clinical trials.

“This multi-institute collaboration involves clinical researchers from institutions across the United States and in Switzerland, all working together to better understand and treat these rare diseases,” said APFED President Dr. Wendy Book. “APFED has a long history of advocating for federal funding to research these conditions, and we are excited to continue participating in CEGIR’s important work with this renewed grant.”

To learn more about CEGIR, visit rdcrn.org/cegir. To learn more about APFED, visit apfed.org.

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About American Partnership for Eosinophilic Disorders (APFED)
The American Partnership for Eosinophilic Disorders (APFED) is a non-profit organization dedicated to patients and their families coping with eosinophilic disorders. APFED’s mission is to embrace, support, and improve the lives of patients and families affected by eosinophil-associated diseases through education and awareness, research, support, and advocacy. www.apfed.org

About Eosinophil-associated Diseases
Eosinophils are a type of white blood cell that helps our immune systems fight off infections and parasites. They also play a role in allergic reactions and inflammatory processes.

When a person has symptoms and an elevated number of eosinophils in their tissues, organs, and/or bloodstream, without a known cause, he/she may have an eosinophil-associated disease. These conditions are further characterized by the areas of the body in which the eosinophils have accumulated.

A few examples include the esophagus (eosinophilic esophagitis), stomach (eosinophilic gastritis), or the lungs (eosinophilic asthma).

Symptoms of eosinophil disease may vary depending on the area of the body affected, and by age. Patients often embark on a long, frustrating journey seeing many different specialists before a diagnosis is made. Although not commonly life-threatening, these chronic diseases require lifelong treatment and can cause debilitating symptoms. Most subsets of eosinophil-associated disease do not yet have an FDA-approved pharmaceutical indicated for treatment.

Related Links:

Digestive Disease Week® (DDW) 2019

APFED is onsite this weekend at Digestive Disease Week® (DDW) in San Diego. This event brings together doctors and researchers in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. The meeting showcases more than 5,000 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. More information can be found at www.ddw.org.

A variety of talks and abstracts on eosinophilic gastrointestinal diseases were presented at the meeting. Below we summarize a few of the highlights:

Sessions and Abstracts

  • “Prospective Evaluation of a Novel, Endoscopic Activity Assessment System for Eosinophilic Gastritis,” presented by Dr. Ikuo Hirano. This research
    highlighted data from a study conducted by CEGIR aimed to evaluate endoscopic features in both children and adults with eosinophilic gastritis (EG) that were enrolled in CEGIR’s OMEGA study so that an endoscopic scoring instrument — the Eosinophilic Gastritis Endoscopic Reference System (EG-REFS)– could be developed. This scoring system is important because an instrument to validate outcome measures in EGID is needed to help advance clinical trials for EGID.
  • “Close Follow-up is Associated with Fewer Stricture Formation and Results in Earlier Detection of Histological Relapse in the Long-term Management of Eosinophilic Esophagitis,” presented by Dr. Thomas Grueter. In this research study, the authors concluded that they advocate for “regular assessment of disease activity in order to detect
    relapsing disease as early as possible, which might minimize the risk for disease complications.”
  • “Eosinophilic Esophagitis-Like Disease with Lack of Significant Esophageal Eosinophilia: Description of a New Disease Entity,” presented by Dr. Thomas Grueter. This abstract described a new disease entity that is like eosinophilic esophagitis, but lacks significant eosphageal eosinophilia. “EoE-like disease is a new entity with distinct (immuno)-histological features, but shared clinical features with EoE,” concluded the authors of the study. “…Despite the absence of significant eosinophilia, considerable disease activity can be observed. Follow-up is warranted to assess for progression to EoE.”
  • “Oral Viscous Budesonide Versus Swallowed Fluticasone Inhaler for Initial Treatment of Adolescents and Adults with Eosinophilic Esophagitis: A Randomized, Double-Blind, Double-Dummy Clinical Trial,” presented by Dr. Evan Dellon. This research set out to determine whether oral viscous budesonide is more effective than swallowing fluticasone from a multi-dose inhaler (MDI) to lower eosinophil counts in the esophagus and improve symptoms of dysphagia for initial treatment of EoE . This research showed both treatments significantly decreased the eosinophil counts, improved the symptoms of dysphagia as well as endoscopic features. “A swallowed slurry was not superior to MDI, indicating either OVB or fluticasone MDI are acceptable choices for EoE therapy,” the authors of the abstract noted.
  • “Functional Luminal Imaging Probe Provides an Accurate Assessment of Esophageal Diameter in Patients with Eosinophilic Esophagitis Undergoing Dilation,” presented by Dr. Ronak Patel. This research involved a cohort of 30 adult patients who have EoE, with an average age of 40 (70% being male), found that this probe when used during endoscopy may improve the accuracy of identifying strictures, which in turn would help improve decision making when it comes to therapeutic dilation.
  • “Eosinophilic Esophagitis and Gastroenteritis 2019: State-of-the-Art,” presented by Dr. David Katzka underscored the key themes of EGID research presented at DDW and delved into characteristics and challenges of eosinophilic gastroenteritis.
  • A session titled “Scientific Advances in Eosinophilic Esophagitis and Gastroenteritis” brought together attendees to discuss how genetic analyses are helping to improve scientific understanding of EGIDs, the importance of novel mediators in EoE pathogenesis, as well as the role of dendritic and epithelial cells in EoE. Speakers included Drs. Tetsuo Shoda, Eunice Odiase, Edaire Cheng, Elizabeth Jensen, Amanda Muir and Joshua Wechsler.
  • A clinical symposium held on Saturday titled “Eosinophilic Esophagitis: What’s New and What to Do” featured lectures given by Drs. Fouad Moawad, Javier Molina-Infante, Alex Straumann, and Mirna Chehade. Attendees learned about factors associated with increasing prevalence of EoE, PPI therapy management in EoE, and treatment options for EoE and EGE patients who don’t respond to steroids.
  • An abstract authored by Evan Dellon, et al, highlighted results from the interim dataset from patients with EG/EGE from the screening phase of a Phase 2 study of AK002, a therapeutic antibody that targets Siglec-8, which is an inhibitory receptor found on the surface of eosinophils and mast cells. The authors concluded that, “…in addition to GI eosinophilia, [mast cell] counts were markedly and consistently elevated in gastric and duodenal tissue in symptomatic patients with overlapping EG and EGE.” These findings suggest that both mast cells and eosinophils have a pathogenic role in EGIDs. “Treatments for EGIDs may need to target both cell types for optimal effect,” the authors noted.
    “Mast cells in addition to eosinophils are markedly elevated at baseline in patients with eosinophilic gastritis and/or gastroenteritis.” Dellon ES, Peterson KA, Genta RM, et. al.
  • Investigators shared their findings from aggregated Electronic Health Record data from 26 major integrated healthcare systems in the U.S. from 1999-2018. “We found that individuals with CD [Crohn’s Disease] are nearly four times more likely to have a diagnosis of EoE compared to individuals without CD,” the authors write. They also noted that further research is needed to confirm their findings.

   “Prevalence of eosinophilic esophagitis in Crohn’s Disease in the United States between 2013 and 2018: A population-based national study.” Mansoor E, Sheriff MZ, Saleh MA, et al.

  • Researchers at the University of Pennsylvania and the Children’s Hospital of Philadelphia set out to examine the burden of endoscopy on adult patients with EoE at their tertiary referral center between 2007-2017. Their findings showed a total of 1,044 patients with EoE had at least one upper endoscopy over the course of the 10-year period that they analyzed. “The average number of endoscopies done per patient was 3.4 with a maximum of 13 endoscopies,” the authors wrote. The most common reasons for patients needing multiple endoscopies were esophageal strictures requiring multiple dilations (38%) and disease not responding to medical and dietary management (62%). “These procedures can result in significant financial burden on patients as well as the health-care system,” the authors noted as part of their conclusion. “Findings support the need to develop alternatives to endoscopy in the treatment and diagnosis of EoE.”

   “The endoscopic burden of eosinophilic esophagitis over a 10-year-perioid.” Gluckman C, Falk GW, Muir AB, Benitez A, Lynch KL.

CEGIR-related work:
Highlights of research conducted by the Consortium for Eosinophilic Gastrointestinal Disease Researchers (CEGIR):

  • In a retrospective study to assess the prevalence of eosoinophilic colitis (EC) at five pediatric and three adult hospitals over the last 3-10 years, investigators found that EC may be over-diagnosed using pediatric ICD coding “In summary, we found that EC is less common than previously described, suggesting over-diagnosis of EC …Establishing clinical-histopathologic guidelines and distinct ICD codes for EC will allow for better estimation of disease prevalence and will inform future clinical and translational studies involving EGIDs,” the authors note.

    “Over Estimation of the Prevalence of Eosinophilic Colitis with Reliance on a Single Billing Code.” Muir AB, Jensen ET, Wechsler, et al

  • Using data collected from the Contact Registry that was established by CEGIR and associated patient advocacy groups, including APFED, investigators analyzed patient reported symptoms and co-morbidities in EoE relative to non-EoE EGIDs: eosinophilic gastritis, eosinophilic gastroenteritis and eosinophilic colitis. The study authors noted that weight loss and gastroparesis was more frequently reported by those with non-EoE subsets of EGID, and that these patients were more likely to report higher frequency of nausea, stomach pain, diarrhea, constipation, bloating, fatigue, feeling of isolation, and deep muscle or joint pain. “Significant differences exist in the symptoms and co-morbidities experienced between those with EoE versus a non-EoE EGID(s), with more severe symptoms and higher frequency of comorbidieis experienced by those with non-EoE EGIDs,” the authors write. “Additional investigation is needed to elucidate the factors that may contribute to the high disease burden of these poorly-understood conditions.”
    “High patient-reported disease burden for eosinophilic gastrointestinal disorders.” Jensen ET, Abonia JP, Aceves SS, et al.
  • Researchers set out to develop and validate a diagnostic panel for eosinophilic gastritis (EG) diagnosis and management. And to help better understand that pathogenesis of this condition. The authors noted that preliminary evidence showed that this diagnostic panel score better correlated with symptoms then eosinophil counts for patients with intermediate tissue eosinophil levels (≥30 HPF).
    “Development of the eosinophilic gastritis molecular diagnostic panel.” Shoda T, Wen T, Caldwell JM, et al.
  • CEGIR investigators aimed to prospectively evaluate endoscopic features in patients with eosinophilic gastritis so that there is a better understanding of endoscopic manifestations and develop a scoring instrument. “Endoscopic features were prospectively recorded in real-time using a classification and grading system specifically developed for EG,” the study authors note. “A strong and significant correlation between [these] scores and physician global assessment of endoscopy severity was demonstrated.
    “Prospective evaluation of a novel, endoscopic activity assessment system for eosinophilic gastritis.” Hirano I, Collins, MH, King E, et al.

APFED Executive Director Mary Jo Strobel; Dr. David A. Leiman, Assistant Professor of Medicine, Division of Gastroenterology, Duke University Medical Center; and Dr. Rajitha Venkatesh, Pediatric Gastroenterologist, Duke Children’s Hospital & Health Center

Dr. Alain Benitez, Clinical Research Program Manager, Suzi and Scott Lustgarten Center for GI Motility at Children’s Hospital of Philadelphia and Dr. Amanda Muir,  Pediatric Gastroenterologist in the Division of Gastroenterology, Hepatology and Nutrition at Children’s Hospital of Philadelphia.

Dr. Mirna Chehade, Associate Professor of Pediatrics and Medicine at the Icahn School of Medicine at Mount Sinai and at the Mount Sinai Kravis Children’s Hospital and APFED Executive Director Mary Jo Strobel