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iTTP: Don't Wait for Test Results
Patients with immune thrombotic thrombocytopenic purpura (iTTP) often die quickly from the disease if the diagnosis is initially missed, a hematologist warned colleagues during a recent webinar on rare and ultrarare blood disorders sponsored by the Association of VA Hematology/Oncology (AVAHO). Clinicians should treat it immediately if it is suspected instead of waiting for confirmatory tests.
The stakes are high, with mortality reaching > 90% in untreated cases, said Yazan Abou-Ismail, MD, of the University of Utah Huntsman Cancer Institute.
“Early recognition and prompt treatment are crucial for survival outcomes,” Abou-Ismail said.
iTTP has an incident rate of 2 to 6 cases per 1,000,000 and is triggered when antibodies target the ADAMTS13 enzyme. This causes platelets to clump together, leading to clots in smaller blood vessels.
Diagnosing iTTP is difficult: “It can have an acute onset, or it could be insidious, meaning it could be slower, perhaps with symptoms originating a few weeks prior to recognition,” Abou-Ismail said.
Between 40% and 80% of patients present with neurologic problems such as headaches, confusion, vision changes, seizures, or stroke-like symptoms. Five additional symptoms—fever, hemolytic anemia, thrombocytopenia, and renal and neurologic dysfunction—are known as a classic diagnostic pentad. In fact, Abou-Ismail said < 10% of iTTP patients have this quintuplet.
Roles for a Test and a Tool
Confirmatory ADAMTS13 tests are recommended, but they can take a week.
“We typically do not wait for the ADAMTS13 result to come back to initiate treatment,” Abou-Ismail said, before adding, “death is most common at initial presentation.”
He advised using the free PLASMIC tool, which provides scores that indicate risk of iTTP.
“It is meant to be used in those with a suspected TMA [thrombotic microangiopathy],” Abou-Ismail said. “It includes 7 components and can identify patients who are at an intermediate risk, high risk, or low risk of severe ADAMTS13 deficiency. Typically, those with a score of ≥ 6 are indicated for empiric treatment until the ADAMTS13 result comes back and either confirms or rules out iTTP.”
Abou-Ismail cautioned, however, that the PLASMIC test is not diagnostic and is not validated in pregnancy.
iTTP Therapy Options
First-line therapy for iTTP includes corticosteroids and therapeutic plasma exchange (PLEX), Abou-Ismail said. PLEX “should be started immediately and empirically if there is a high pretest probability of iTTP based on clinical judgment or tools such as a PLASMIC score.”
VA facilities may not be able to handle PLEX due to the need for proper personnel, equipment and machinery, he said. If so, patients should be referred to a tertiary referral center.
“One thing to keep in mind is that plasma exchange is not entirely benign and can have its own complications,” he said. “They’re not very common, but they can occur in up to 10% to 15% of patients.”
Abou-Ismail also highlighted adjunctive therapy, mainly with rituximab and caplacizumab. Looking beyond immediate treatment, he noted that iTTP survivors can have multiple complications.
“Long-term laboratory and clinical monitoring remain necessary even after clinical remission.”
Abou-Ismail disclosed relationships with Sanofi, Takeda, and Genentech.
Patients with immune thrombotic thrombocytopenic purpura (iTTP) often die quickly from the disease if the diagnosis is initially missed, a hematologist warned colleagues during a recent webinar on rare and ultrarare blood disorders sponsored by the Association of VA Hematology/Oncology (AVAHO). Clinicians should treat it immediately if it is suspected instead of waiting for confirmatory tests.
The stakes are high, with mortality reaching > 90% in untreated cases, said Yazan Abou-Ismail, MD, of the University of Utah Huntsman Cancer Institute.
“Early recognition and prompt treatment are crucial for survival outcomes,” Abou-Ismail said.
iTTP has an incident rate of 2 to 6 cases per 1,000,000 and is triggered when antibodies target the ADAMTS13 enzyme. This causes platelets to clump together, leading to clots in smaller blood vessels.
Diagnosing iTTP is difficult: “It can have an acute onset, or it could be insidious, meaning it could be slower, perhaps with symptoms originating a few weeks prior to recognition,” Abou-Ismail said.
Between 40% and 80% of patients present with neurologic problems such as headaches, confusion, vision changes, seizures, or stroke-like symptoms. Five additional symptoms—fever, hemolytic anemia, thrombocytopenia, and renal and neurologic dysfunction—are known as a classic diagnostic pentad. In fact, Abou-Ismail said < 10% of iTTP patients have this quintuplet.
Roles for a Test and a Tool
Confirmatory ADAMTS13 tests are recommended, but they can take a week.
“We typically do not wait for the ADAMTS13 result to come back to initiate treatment,” Abou-Ismail said, before adding, “death is most common at initial presentation.”
He advised using the free PLASMIC tool, which provides scores that indicate risk of iTTP.
“It is meant to be used in those with a suspected TMA [thrombotic microangiopathy],” Abou-Ismail said. “It includes 7 components and can identify patients who are at an intermediate risk, high risk, or low risk of severe ADAMTS13 deficiency. Typically, those with a score of ≥ 6 are indicated for empiric treatment until the ADAMTS13 result comes back and either confirms or rules out iTTP.”
Abou-Ismail cautioned, however, that the PLASMIC test is not diagnostic and is not validated in pregnancy.
iTTP Therapy Options
First-line therapy for iTTP includes corticosteroids and therapeutic plasma exchange (PLEX), Abou-Ismail said. PLEX “should be started immediately and empirically if there is a high pretest probability of iTTP based on clinical judgment or tools such as a PLASMIC score.”
VA facilities may not be able to handle PLEX due to the need for proper personnel, equipment and machinery, he said. If so, patients should be referred to a tertiary referral center.
“One thing to keep in mind is that plasma exchange is not entirely benign and can have its own complications,” he said. “They’re not very common, but they can occur in up to 10% to 15% of patients.”
Abou-Ismail also highlighted adjunctive therapy, mainly with rituximab and caplacizumab. Looking beyond immediate treatment, he noted that iTTP survivors can have multiple complications.
“Long-term laboratory and clinical monitoring remain necessary even after clinical remission.”
Abou-Ismail disclosed relationships with Sanofi, Takeda, and Genentech.
Patients with immune thrombotic thrombocytopenic purpura (iTTP) often die quickly from the disease if the diagnosis is initially missed, a hematologist warned colleagues during a recent webinar on rare and ultrarare blood disorders sponsored by the Association of VA Hematology/Oncology (AVAHO). Clinicians should treat it immediately if it is suspected instead of waiting for confirmatory tests.
The stakes are high, with mortality reaching > 90% in untreated cases, said Yazan Abou-Ismail, MD, of the University of Utah Huntsman Cancer Institute.
“Early recognition and prompt treatment are crucial for survival outcomes,” Abou-Ismail said.
iTTP has an incident rate of 2 to 6 cases per 1,000,000 and is triggered when antibodies target the ADAMTS13 enzyme. This causes platelets to clump together, leading to clots in smaller blood vessels.
Diagnosing iTTP is difficult: “It can have an acute onset, or it could be insidious, meaning it could be slower, perhaps with symptoms originating a few weeks prior to recognition,” Abou-Ismail said.
Between 40% and 80% of patients present with neurologic problems such as headaches, confusion, vision changes, seizures, or stroke-like symptoms. Five additional symptoms—fever, hemolytic anemia, thrombocytopenia, and renal and neurologic dysfunction—are known as a classic diagnostic pentad. In fact, Abou-Ismail said < 10% of iTTP patients have this quintuplet.
Roles for a Test and a Tool
Confirmatory ADAMTS13 tests are recommended, but they can take a week.
“We typically do not wait for the ADAMTS13 result to come back to initiate treatment,” Abou-Ismail said, before adding, “death is most common at initial presentation.”
He advised using the free PLASMIC tool, which provides scores that indicate risk of iTTP.
“It is meant to be used in those with a suspected TMA [thrombotic microangiopathy],” Abou-Ismail said. “It includes 7 components and can identify patients who are at an intermediate risk, high risk, or low risk of severe ADAMTS13 deficiency. Typically, those with a score of ≥ 6 are indicated for empiric treatment until the ADAMTS13 result comes back and either confirms or rules out iTTP.”
Abou-Ismail cautioned, however, that the PLASMIC test is not diagnostic and is not validated in pregnancy.
iTTP Therapy Options
First-line therapy for iTTP includes corticosteroids and therapeutic plasma exchange (PLEX), Abou-Ismail said. PLEX “should be started immediately and empirically if there is a high pretest probability of iTTP based on clinical judgment or tools such as a PLASMIC score.”
VA facilities may not be able to handle PLEX due to the need for proper personnel, equipment and machinery, he said. If so, patients should be referred to a tertiary referral center.
“One thing to keep in mind is that plasma exchange is not entirely benign and can have its own complications,” he said. “They’re not very common, but they can occur in up to 10% to 15% of patients.”
Abou-Ismail also highlighted adjunctive therapy, mainly with rituximab and caplacizumab. Looking beyond immediate treatment, he noted that iTTP survivors can have multiple complications.
“Long-term laboratory and clinical monitoring remain necessary even after clinical remission.”
Abou-Ismail disclosed relationships with Sanofi, Takeda, and Genentech.
iTTP: Don't Wait for Test Results
iTTP: Don't Wait for Test Results