# Defining Clinical Research Priorities and Strengthening Diversity and Patient-Stakeholder Engagement in Histiocytic Disorders

> **NIH NIH R13** · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · 2024 · $30,000

## Abstract

PROJECT SUMMARY/ABSTRACT
Histiocytic disorders, or histiocytoses, are a family of rare hematologic diseases encompassing the histiocytic
neoplasms (including Langerhans cell histiocytosis, Erdheim-Chester disease, and Rosai-Dorfman disease) and
the hemophagocytic lymphohistiocytosis (HLH) disorders. Histiocytosis affects both adults and children, and
clinical disease is mediated by accumulation of macrophage, dendritic cell, or monocyte-derived cells in
affected tissues. These are orphan diseases with an aggregated prevalence of fewer than 10,000 patients in the
US. Despite important recent advances in histiocytic disorders, key knowledge gaps remain owing to their rarity
and to the absence of collaboratively executed investigation across adults and children and across the disease
subtypes. Specifically, there is a lack of unified approach to observational and natural history studies, as well as
a coordinated approach to the prioritization and implementation of therapeutic trials. Additionally, there is an
urgent need to engage the community of stakeholders in histiocytosis investigation in order to elevate its
patient-centeredness and ensure that research is responsive to the community’s needs. Last, there is a
pressing need for the histiocytosis scientific community to appraise its own composition and to take a deliberate,
inclusive approach towards incorporating diverse perspectives into the vision and execution of future research.
The proposed Histiocytosis Disorders Workshop (HDW) represents an unprecedented gathering of senior and
junior scientists—across adult and pediatric diseases, HLH and histiocytic neoplasms—eager to align priorities
and approaches to research in these diseases. The two-day event will include approximately 50 in-person
diverse attendees including histiocytosis scholars, patients and stakeholders, experts in (a) patient engagement
and patient-reported outcomes, (b) diversity and inclusion, (c) junior career development, and representatives
from NIH, FDA, and PCORI. The workshop will include lectures, moderated panels, and breakout sessions on
the topics of observational studies, therapeutic trials, biomarkers and response assessment, patient reported
outcomes, community engagement, faculty development and inclusiveness, and diversity and inclusion. The
event will be advertised by social media and email to scientific societies and patient and community groups with
emphasis upon inclusion of junior investigators and attendees from diverse communities.
The HDW is timely in (1) its integration of the most current and advanced science related to histiocytosis and (2)
its leveraging the North American Consortium for Histiocytosis (NACHO), a mature and operational consortium
for pediatric histiocytosis to host and implement the meeting. The workshop will be useful to the scientific
community by defining (1) key data elements for observational studies, (2) strategies for prioritizing and
implementing therapeutic trials and (3)...

## Key facts

- **NIH application ID:** 10828093
- **Project number:** 1R13TR004891-01
- **Recipient organization:** ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- **Principal Investigator:** CARL E ALLEN
- **Activity code:** R13 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $30,000
- **Award type:** 1
- **Project period:** 2024-01-01 → 2024-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10828093

## Citation

> US National Institutes of Health, RePORTER application 10828093, Defining Clinical Research Priorities and Strengthening Diversity and Patient-Stakeholder Engagement in Histiocytic Disorders (1R13TR004891-01). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10828093. Licensed CC0.

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