# Development and validation of a disease-relevant patient-reported outcome tool in light chain amyloidosis

> **NIH NIH K23** · MEDICAL COLLEGE OF WISCONSIN · 2021 · $151,688

## Abstract

ABSTRACT
Light chain amyloidosis (AL) is a plasma cell disorder characterized by organ dysfunction from insoluble fibril
deposition derived from clonal light chains. The disease is associated with high early mortality and is treated
with chemotherapies similar to multiple myeloma. Decline in health status is predominant in AL and often the
signature feature at presentation. Even when chemotherapy produces a successful hematologic response,
patients may continue to experience symptoms related to organ (e.g. cardiac, renal, neurologic) dysfunction,
due to deposition of fibrils prior to therapy. Therefore, measurement of PROs, such as patient-reported fatigue
and physical functioning, is a critical, yet underutilized, aspect of the overall outcomes assessment of trials of
AL therapies. The candidate for this mentored patient-oriented research career development (K23) award, Dr.
Anita D'Souza, is a hematology/oncology trained amyloid specialist with demonstrated success in conducting
investigator-initiated clinical trials. As the Scientific Director of the Plasma Cell Disorders Working Committee in
the NIH-funded Center for International Blood and Marrow Transplant Research and member of the Data and
Coordinating Center for the NIH-funded Blood and Marrow Transplant Clinical Trials Network, Dr. D'Souza is
well positioned for a career in developing and executing clinical trials in plasma cell disorders. Her career goal
is to conduct patient-centered amyloid clinical research, where the patient's perspective is a standard and
expected component of clinical trials 1) evaluating new treatments for AL amyloidosis
and 2)
using PROs for
symptom monitoring and management
. To accomplish this goal, Dr. D'Souza requires didactic training and
mentorship in PRO methodology, mentorship in patient-centered research, and mentorship in effective
engagement with patients and patient advocates. In this K23 application, and in the context of providing this
training and mentorship, we propose to develop and evaluate a PRO tool relevant for AL (the AL-PROfile),
utilizing items from the library of NIH PROMIS measures. Specific Aim 1 will determine the most relevant
symptom and functioning domains for patient-centered AL research through literature review and qualitative
interviews with patients, patient advocates, and clinical and research experts, select items from the PROMIS
item libraries to comprise the AL-PROfile, and confirm item comprehension in cognitive interviews with
patients; Aim 2 will evaluate the psychometric measurement properties of the AL-PROfile, including convergent
and discriminant validity, known groups validity, internal consistency, test-retest reliability,
and responsiveness
to change; and Aim 3 will track PROs longitudinally in newly diagnosed patients in clinical care. Dr. D'Souza's
training plan incorporates didactics, mentoring by a multi-disciplinary team, and research experience that will
allow her to augment her expertise in AL clinical tri...

## Key facts

- **NIH application ID:** 10139092
- **Project number:** 5K23HL141445-03
- **Recipient organization:** MEDICAL COLLEGE OF WISCONSIN
- **Principal Investigator:** Anita D'Souza
- **Activity code:** K23 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $151,688
- **Award type:** 5
- **Project period:** 2019-05-01 → 2023-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10139092

## Citation

> US National Institutes of Health, RePORTER application 10139092, Development and validation of a disease-relevant patient-reported outcome tool in light chain amyloidosis (5K23HL141445-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10139092. Licensed CC0.

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