Adzynma safely cuts relapse risk in TTP patients, new study finds
Therapy may protect against long-term organ damage, improve life quality
The preventive treatment Adzynma (recombinant human ADAMTS13) is safe and effective at reducing disease relapses for people with congenital thrombotic thrombocytopenic purpura (TTP), and may help to prevent long-term organ damage and improve quality of life, a French study has found.
“Long-term organ complications … highlight the need for personalized follow-up and tailored prophylactic strategies,” researchers wrote.
The study, “Optimizing ADAMTS13 prophylaxis to reduce relapse and organ failure in congenital thrombotic thrombocytopenic purpura,” was published in Blood Advances.
Study design and patient demographics
In TTP, blood clots form in small vessels, leading to organ damage. Congenital TTP is caused by mutations that result in a severe deficiency of ADAMTS13, an enzyme that prevents the formation of unneeded blood clots.
While episodes of TTP can be life-threatening, less is known about long-term complications and how well preventive treatments work. To learn more, researchers carried out a study (NCT00426686) that drew on 25 years of data from 88 patients (83 families) diagnosed with congenital TTP. Of note, some of the researchers had financial links to pharmaceutical companies, including Takeda, which markets Adzynma.
The patients were divided into three groups based on when the disease first appeared: childhood (42 patients), pregnancy (33), or adulthood (13). Treatment included plasma infusions to supply functional ADAMTS13 and, since 2020, enzyme replacement therapy with Adzynma, which contains a recombinant (lab-made) version of ADAMTS13.
All patients had low counts of red blood cells and platelets, which are consumed during blood clotting. Emergency treatment included blood transfusions, plasma infusions, or plasma exchange, in which a patient’s own plasma (the part of blood that doesn’t contain cells) is removed and replaced by healthy plasma. Some patients had received immunosuppressants before being diagnosed with TTP.
Children usually developed TTP very early, with a median age at diagnosis of 2 years. They often experienced relapses, when symptoms worsen or reappear after a period of remission. Many children developed mental health problems (24%) and high blood pressure (19%).
In women who first showed symptoms of TTP during pregnancy, the disease relapsed only during pregnancy. However, if left untreated during pregnancy, both mother and baby might experience complications.
Adults diagnosed later in life, at a median age of 55, often experienced complications during follow-up. About two-thirds (69%) had high blood pressure, and nearly half (46%) had mental health problems, including depression (31%), anxiety (31%), and post-traumatic stress (15%). Cognitive problems were also common (15%).
Despite long-term plasma infusions, many patients continued to show signs of organ dysfunction.
“This strategy remains associated with substantial limitations, including allergic reactions, volume overload, and the logistical burden of regular infusions, particularly in children,” the researchers wrote.
Of the 39 patients who received treatment with Adzynma, 36 switched to regular prophylaxis every one or two weeks. Side effects were mild and included mostly headaches and gastrointestinal symptoms. The use of Adzynma rapidly increased ADAMTS13 in the blood, usually within one hour of the infusion.
Over a median follow-up of five months, Adzynma reduced the risk of relapses by 52% compared with no treatment. In childhood-onset TTP, Adzynma worked as well as plasma infusions in preventing relapses, but had fewer side effects.
“By reducing the burden of care,” prophylactic treatment with Adzynma “represents a safe and promising first-line option that should help [in] reducing long-term organ damage and improving quality of life,” the researchers wrote. “As the therapeutic landscape evolves, future efforts should focus on personalized management.”
About the Author
