# Mycobacterium avium complex Core Outcomes Research (MACCOR)

> **NIH NIH K23** · OREGON HEALTH & SCIENCE UNIVERSITY · 2024 · $169,199

## Abstract

PROJECT SUMMARY/ABSTRACT
Pulmonary Mycobacterium avium complex (MAC) is a chronic infection and airway inflammatory disease with
systemic manifestations affecting multiple aspects of our patients' lives. Similar to other chronic inflammatory
diseases, such as rheumatoid arthritis, the impact of pulmonary MAC disease (pMACd) varies between
individuals with regards to presentation, course and response to therapy. Lifelong management is often
required, as recurrence is common. The treatment burden is high with patients typically taking 3-5 antibiotics
for 18-24 months. In clinical practice, sputum culture results, radiographic findings, and symptoms drive
treatment decisions. While conversion of sputum culture to negative implies microbiologic success, it does not
imply infection eradication and it is inadequate to assess the full impact of this disease on our patients' lives.
Importantly, the US Food and Drug Administration does not consider culture conversion an adequate endpoint
in therapeutic trials for pMACd. As this disease has become recognized to be increasing in incidence and its
tremendous unmet need for antibiotics highlighted, a pipeline of new therapeutics for this orphan disease has
recently materialized. However randomized clinical trials are currently stymied by a lack of validated outcome
measures. Objective measures of function, health-related quality of life, radiographic changes and biomarkers
are needed as clinical outcomes. Further, the heterogeneity of this disease's natural history is reflected by the
fact that culture, radiography, and symptomatology often do not change together. A combined outcome
measure along several domains (symptoms, radiologic, etc.) would better capture disease activity and could be
useful both clinically as well as in clinical trials of new therapeutics. Currently, there are no validated outcome
measures in pMACd and potential outcomes are not measured consistently across all studies. In order to
facilitate development of a future combined disease activity measure, this proposal aims to identify a set of
core outcome domains and the best available instruments for a minimum core outcome measurement set that
can be consistently collected across future clinical trials. First, we will identify a set of core outcome domains
for pMACd essential to patients, caregivers, patient advocates, clinical experts, and researchers through an
international consensus process using the Delphi methodology. Second, we will evaluate the impact of
pulmonary MAC treatment on various available measurement instruments that represent the identified core
outcome domains, by utilizing available clinical datasets. Third, we will identify a minimum core outcome
measurement instrument set essential for future pMACd clinical trials through the international Delphi
consensus process. This will enable comparisons between clinical trials and meta-analyses, both important in
a rare disease such as pulmonary MAC. The results of this proj...

## Key facts

- **NIH application ID:** 10894740
- **Project number:** 5K23HL161495-03
- **Recipient organization:** OREGON HEALTH & SCIENCE UNIVERSITY
- **Principal Investigator:** Cara Varley
- **Activity code:** K23 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $169,199
- **Award type:** 5
- **Project period:** 2022-08-15 → 2027-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10894740

## Citation

> US National Institutes of Health, RePORTER application 10894740, Mycobacterium avium complex Core Outcomes Research (MACCOR) (5K23HL161495-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10894740. Licensed CC0.

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