# MIDAS: MIcroangiopathy, endothelial Damage in Adults undergoing Stem cell transplantation

> **NIH NIH R01** · OHIO STATE UNIVERSITY · 2024 · $728,230

## Abstract

MIDAS: Microangiopathy, endothelial Damage in Adults undergoing Stem cell transplantation
ABSTRACT
Hematopoietic cell transplant-associated thrombotic microangiopathy (HCT-TMA) is a clinical diagnosis based
on consensus criteria and associated with high mortality rates (80%). Approximately 9,000 hematopoietic cell
transplant (HCT) procedures are performed in the U.S. annually, and reported frequencies of HCT-TMA are
highly variable due to lack of routine screening. HCT-TMA is a morbid and potentially life-threatening
complication of HCT including microangiopathic hemolytic anemia, renal dysfunction and neurological
symptoms. The initiating event of HCT-TMA appears to be endothelial injury, and extensive data indicate decline
in endothelial health as people age suggesting that HCT-TMA might be more frequent or severe in older persons.
Preliminary data indicate a bimodal distribution of HCT-TMA in adults, with a peak early after transplantation,
which is well described in children, but a second later peak 3-6 months after transplant, sometimes associated
with a flare of graft versus host disease (GVHD) during taper of immune suppression. There is no reported
prospective study of HCT-TMA in adults thus risk factors and outcomes are currently unknown. This is a key gap
in current knowledge and we plan to address this gap in our proposal. The need for a prospective adult cohort
study of HCT-TMA is urgent as older patients are increasingly eligible for HCT. This study will define clinical
phenotypes, risk factors, and possible therapeutic strategies for HCT-TMA. Our overarching hypothesis is that
“The etiology and risk factors for HCT-TMA are different in adults than children, and that these differences
importantly modify potential diagnostic and therapeutic strategies”. We propose to identify strategies that will
define HCT recipients with increased susceptibility to HCT-TMA occurring early after transplantation, and later
after establishment of GVHD. Identifying endothelial injury occurring post-HCT at the earliest possible time will
allow for prompt clinical intervention and interruption of the cycle of endothelial injury and complement activation.
The centers participating in this study are ideal for this work because they are large transplant centers with a
strong track record of successful clinical research and study enrollment with a long-standing interest in HCT-
TMA, evidenced by previous publications in the area. We will use our prospectively generated, well-annotated
clinical database to test hypotheses regarding pathophysiology, for example, that GVHD is a major contributor
to HCT-TMA by examining clinical risk factors and biomarkers of endothelial injury. We will measure the financial
cost of HCT-TMA, late organ toxicity and will formulate and test a predictive index for HCT-TMA to target
monitoring and treatment to highest-risk individuals. In summary, this study will provide essential data to identify
persons at highest risk of HCT-TMA t...

## Key facts

- **NIH application ID:** 10894946
- **Project number:** 5R01HL153723-05
- **Recipient organization:** OHIO STATE UNIVERSITY
- **Principal Investigator:** Sumithira Vasu
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $728,230
- **Award type:** 5
- **Project period:** 2020-08-20 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10894946

## Citation

> US National Institutes of Health, RePORTER application 10894946, MIDAS: MIcroangiopathy, endothelial Damage in Adults undergoing Stem cell transplantation (5R01HL153723-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10894946. Licensed CC0.

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