Screening Is Critical Component of TA-TMA Management

Christopher E. Dandoy, MD, MSc, assistant professor in the University of Cincinnati Department of Pediatrics, Cincinnati Children's Hospital

Christopher E. Dandoy, MD, MSc

Pediatric stem cell transplant recipients should be screened for transplant-associated thrombotic microangiopathy (TA-TMA), which, if adequately diagnosed and treated, can lead to improved outcomes, said Christopher E. Dandoy, MD, MSc.

"We recommend obtaining patients’ complete blood counts for the first month posttransplant as well as [watch their] renal profile by looking for kidney injury on a daily basis. On top of that, [providers] should be looking at lactate dehydrogenase, urine for proteinuria, and closely monitoring patients’ blood pressure," said Dandoy.

In an interview with OncLive, Dandoy, an assistant professor in the University of Cincinnati Department of Pediatrics, Cincinnati Children’s Hospital, discussed the clinical features of TA-TMA and the importance of close monitoring and intervention for patients at risk of developing the disorder.

OncLive: What are the current treatments for patients with TA-TMA?

Dandoy: TA-TMA is most commonly treated with eculizumab (Soliris). Some new drugs are coming up in the market that, in the next few years, will be available to patients.

What are the challenges of managing patients with TA-TMA?

The primary challenge right now is diagnosing and identifying patients who have TA-TMA. We recommend screening for TA-TMA.

What are the clinical features of TA-TMA?

TA-TMA affects the endothelium, which [lines] our body. The organs that are primarily affected are the kidneys, which can lead to proteinuria. TA-TMA can affect the lungs, the heart, the gut, the skin, and the central nervous system.

We know that patients who develop TA-TMA have worse overall outcomes. These patients have a higher fatality rate than those who do not develop TA-TMA. We believe that if TA-TMA is adequately diagnosed and treated, it leads to improved outcomes and improved survival.

It's important to screen for [TA-TMA] and be aware of it. Patients have severe [TA-TMA] by way of proteinuria, terrible hypertension, or activated complement. We've shown that it’s important to treat [these patients] in a timely manner.

What efforts have you been involved with regarding treatment strategies in this space?

We collaborated with 13 pediatric institutions throughout the United States. These institutions instigated this screening [modality] that we recommended. We did a retrospective analysis of approximately 614 patients to look for the incidence, risk factors, and outcomes of patients who developed TA-TMA.

We saw that roughly 18% of pediatric stem cell transplant recipients developed TA-TMA or were diagnosed with TA-TMA. Those patients had significantly higher nonrelapse-related mortality. These patients were in the intensive care unit for longer periods of time, were hospitalized for longer periods of time, had a higher rate of developing bloodstream infections, and required renal replacement therapy as well as intubation. All of these things were worse in patients who developed TA-TMA.

We concluded that TA-TMA affects a larger percentage of patients than we thought and that it is associated with worse outcomes. We have to screen for it and know that it does exist. If you do find it, you need to treat patients appropriately.

If you screen for TA-TMA and see it, you can treat it. If you don't feel comfortable or don't know how to treat it, reach out to folks that have done it before. We're a pretty welcoming and collegial group of folks. We're open to helping patients out throughout the country and the world. [You can always] reach out to other folks who have a little bit more experience if you're uncomfortable [treating it].