# 2/2 A comparison of individualized vs. weight based protocols to treat vaso-occlusive episodes in Sickle Cell Disease (COMPARE VOE)

> **NIH NIH U24** · DUKE UNIVERSITY · 2020 · $1,221,928

## Abstract

Abstract
Sickle cell disease (SCD) is a genetic disorder and the most common hemoglobin disorder in the United States
(U.S.). It affects an estimated 90,000-100,000 Americans and was ranked as the fifth most common principal
diagnosis for Medicaid's super-utilizer hospital stays. Painful vaso-occlusive episodes (VOE), a new onset of
pain (7/10 or greater) for at least four hours for which there is no other explanation than vaso-occlusion, are the
most common manifestation of SCD experienced by patients and the most common reason for emergency
department (ED) visits. There is no standard, evidence based approach to treating painful VOE. In 2014 the
National Heart, Lung, and Blood Institute (NHLBI) released recommendations for the “Management of Sickle
Cell Disease” where recommendations to treat VOE were based on consensus panel expertise due to lack of
evidence. Recommendations included the use of a patient specific protocol (specific agents and doses for an
individual patient) or a standard VOE analgesic protocol, where the standard VOE protocol was not defined.
We implemented a weight-based analgesic protocol as a “standard” VOE protocol and conducted a pilot
randomized clinical trial (RCT) at two EDs with 52 patients comparing the patient-specific and weight-based
analgesic protocols; results were encouraging, however, limited due to small numbers of patients and sites.
We propose a Phase III randomized and single-blinded (with subject blinded) clinical trial to test the hypothesis
that the patient-specific analgesic protocol is clinically superior to the weight-based analgesic protocol for
treating SCD patients with an ED visit due to VOE. This five-site RCT will randomize 460 adult SCD patients to
one of the two protocols in order to have the target of 230 patients with an ED visit for VOE during the 24
month enrollment period. A sample size of 230 patients will provide 90% power for detecting a 14 mm clinically
significant difference between the two protocols in the primary endpoint, pain score reduction from arrival in the
ED to discharge by using a 0-100 mm visual analog scale. Secondary outcomes include ED length of stay,
hospitalization, seven day return ED visits and hospitalizations and we will monitor side effects and safety. A
Clinical Coordinating Center (CCC; Paula Tanabe, PI) and a Data Coordinating Center (DCC; Huiman
Barnhart, PI) will manage this trial. This experienced multi-specialty team will deliver the integrated services
vital to the trial, including clinical and operational leaderships, site management, statistical analysis and data
management. High level evidence generated by this trial will shift the paradigm on treating VOE in the ED and
decreasing the pain of SCD patients suffering from this devastating complication.

## Key facts

- **NIH application ID:** 10021701
- **Project number:** 5U24HL137907-03
- **Recipient organization:** DUKE UNIVERSITY
- **Principal Investigator:** Huiman X Barnhart
- **Activity code:** U24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,221,928
- **Award type:** 5
- **Project period:** 2018-09-07 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10021701

## Citation

> US National Institutes of Health, RePORTER application 10021701, 2/2 A comparison of individualized vs. weight based protocols to treat vaso-occlusive episodes in Sickle Cell Disease (COMPARE VOE) (5U24HL137907-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10021701. Licensed CC0.

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