Cablivi boosts ICU survival odds for people with severe iTTP in analysis
Despite severe symptoms, patients showed improvement during hospital stay
Most people with the rare blood disorder immune-mediated thrombotic thrombocytopenic purpura (iTTP) recover well after admission to the intensive care unit (ICU), and treatment with the medication Cablivi (caplacizumab-yhdp) appears linked to improved survival rates.
The findings, drawn from a retrospective analysis of patients treated in German hospitals, highlight the favorable prognosis for iTTP patients despite often experiencing severe neurological or heart-related symptoms.
Most patients arrived in the ICU during their first episode of the disease. Although standard therapy with plasma exchange (PEX) and corticosteroids was used in nearly all episodes, none of the Cablivi-treated episodes resulted in death, and PEX was needed for a shorter time.
Cablivi may reduce ICU deaths
The study, “Characteristics and Management of Patients With Immune Thrombotic Thrombocytopenic Purpura Admitted to the Intensive Care Unit: A Multicenter Retrospective Analysis,” was published as a correspondence in the American Journal of Hematology.
iTTP, also known as acquired TTP, occurs when antibodies mistakenly attack ADAMTS13, an enzyme that normally breaks down von Willebrand factor (vWF). Without enough ADAMTS13, vWF binds to platelets, the clot-forming fragments in blood, causing them to clump when they shouldn’t. As clots form in small blood vessels, platelets are used up, red blood cells are destroyed as they squeeze past the clots, and organs are damaged by the reduced blood supply, leading to a wide range of possible symptoms.
Standard TTP treatment usually relies on PEX, a procedure that removes the patient’s plasma, the acellular part of the blood containing the harmful antibodies, and replaces it with donor plasma. Corticosteroids are added to help suppress the abnormal immune response.
Cablivi, which prevents vWF from binding to platelets, and rituximab, which targets the immune cells that produce disease-driving antibodies, have been shown to speed recovery and reduce relapses, though recommendations for their use remain inconsistent.
Because iTTP can worsen suddenly, many patients experience severe symptoms that require immediate intervention, meaning treatment often begins in ICU settings. Yet little is known about how these patients are managed once they reach the ICU or what their outcomes are, the researchers wrote.
To help fill that gap, the team analyzed 98 iTTP episodes in 95 adults treated in ICUs across 10 hospitals in Germany between 2013 and 2024. Most were women (66.3%), and the median age was 44. In nearly three-quarters of episodes (73.5%), the ICU stay occurred during a first attack of the disease rather than a relapse.
Reasons for ICU admission
Although the need for close monitoring was the most common reason for ICU admission, documented in 83 episodes (84.7%), neurological symptoms also frequently prompted transfer, occurring in 49 of 98 episodes (50%). Heart-related issues were reported in 20 episodes (20.4%).
Once in the ICU, treatment closely followed current practice patterns. In nearly all episodes (94.9%), corticosteroids were given, and in most (86.7%), PEX was also administered. Cablivi was used in 58 episodes (59.2%), while rituximab was given in 33 episodes (33.7%). In 10 episodes (10.2%), Cablivi was administered without PEX.
Complications during ICU care were not uncommon. Neurological complications occurred in 19 episodes (19.2%), infections in 17 episodes (17.3%), and heart-related complications in 15 episodes (15.3%). Bleeding events were documented in 12 episodes (12.2%), with half classified as major bleeds.
Several patients required intensive supportive care. Vasopressors, medications used to raise blood pressure, were needed in 15 episodes (15.3%), and 14 episodes (14.3%) required mechanical ventilation to assist with breathing. Renal replacement therapy, which can include dialysis, was required in five episodes (5.1%). Cardiopulmonary resuscitation was needed in nine episodes (9.2%).
Despite the severity of illness, most patients improved during their ICU stay. The median ICU stay was three days, during which platelet counts rose and markers of red blood cell damage fell. Overall, seven episodes (7.1%) ended in death, typically occurring shortly after ICU admission.
A notable difference emerged when outcomes were compared by treatment approach. While no deaths occurred among the 58 episodes where Cablivi was used, four patients died among the 37 episodes (10.8%) in which Cablivi was not used. Patients who received this therapy also required a median of one day less of PEX, although disease severity at admission and clinical status at discharge were largely similar between the two groups.
“The present analysis demonstrated favorable ICU outcomes for iTTP patients,” the researchers concluded.
They cautioned, however, that differences in initial disease characteristics between the groups, as well as the more frequent use of rituximab in the Cablivi group, might have influenced the results.
About the Author
