Switching to Adzynma slashes hospital stays in congenital TTP
Real-world data shows enzyme therapy outperforms fresh frozen plasma
Switching from treatment with fresh frozen plasma (FFP) to Adzynma (ADAMTS13, recombinant-krhn) can help people with congenital thrombotic thrombocytopenic purpura (TTP) avoid hospital stays, a study from Poland suggests.
“This real-world multicenter study provides compelling evidence for the clinical benefits of switching from FFP to [Adzynma] in Polish patients with congenital TTP,” the researchers wrote. The findings were reported in a letter to the editor titled, “Real-world safety and efficacy of rADAMTS13 prophylaxis in congenital thrombotic thrombocytopenic purpura: experience from Polish patients previously treated with fresh frozen plasma,” which was published in Haematologica.
TTP is marked by a deficiency of ADAMTS13, an enzyme that helps regulate blood clotting. Its congenital form is caused by mutations in the gene that provides instructions for making the enzyme.
Preventive treatment for congenital TTP has long relied on FFP. Plasma is the noncellular portion of blood that contains several proteins and enzymes, including ADAMTS13. Administering FFP from healthy donors can help replenish the missing enzyme to control TTP symptoms.
Adzynma is an enzyme replacement therapy that delivers a functional version of ADAMTS13 to the body. Administered through an injection into the bloodstream, it was approved for congenital TTP in the U.S. in 2023 and in Europe in 2024.
Decline in hospital stays after switch to Adzynma
Here, scientists reported on the outcomes of 23 people with congenital TTP — 16 adults and seven children — who switched from FFP to Adzynma after the new treatment became available through compassionate use. Most of the patients received Adzynma every other week. The researchers compared outcomes in the years immediately before and after the switch.
The results showed the mean number of hospitalizations decreased from more than 17 on FFP to less than one on Adzynma. Consistently, in the year patients were on FFP, they spent nearly 25 days in the hospital on average, whereas in the year they were on Adzynma, the mean time spent in the hospital was less than a day.
“The number of hospitalizations — particularly those related to disease exacerbation — declined significantly in both adults and children,” the researchers wrote. “For pediatric patients, this translated into fewer missed school days and reduced burden on caregivers, underscoring the broader benefits of [Adzynma] beyond [blood-related] parameters.”
The mean number of TTP disease events also was lower with Adzynma and it was better than FFP at increasing hemoglobin levels and platelet counts. Hemoglobin is the oxygen-carrying protein in red blood cells, while platelets are the cell fragments that help blood clot.
“The observed improvements in laboratory parameters, reduction in acute, subacute, and isolated TTP events, and decreased hospitalization rates collectively highlight the effectiveness and safety of [Adzynma] in both adult and pediatric patients,” the researchers wrote.
Safety data also generally favored Adzynma. No side effects related to it were reported while patients were on the therapy, whereas more than half of those with available data had allergic reactions or other side effects related to FFP.
The findings “provide real-world support for the use of [Adzynma] as an effective and well-tolerated prophylactic therapy for congenital TTP, offering clear advantages over traditional FFP-based regimens,” the researchers wrote.
Adzynma is sold by Takeda Pharmaceuticals, which had no involvement in the study.
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