- N Engl J Med 2022; 387:237-247
Nine of 10 patients with hemophilia B who received a liver-directed adeno-associated viral vector containing a hyperactive variant of factor IX maintained factor IX activity for up to 42 months. Thrombosis developed in 1 patient.
- N Engl J Med 2022; 387:160-166
Inhibitors of proximal and terminal complement cascades can often control red-cell hemolysis associated with paroxysmal nocturnal hemoglobinuria, but some patients have breakthrough hemolysis.
- N Engl J Med 2022; 387:88-90
In 69 patients with vaccine-induced immune thrombotic thrombocytopenia caused by anti–PF4 antibodies, subsequent Covid-19 infection or receipt of an mRNA-based vaccine did not induce VITT recurrence.
- N Engl J Med 2022; 386:1986-1997
Tranexamic acid is an antifibrinolytic drug that reduces bleeding in patients undergoing cesarean section or cardiac surgery. In this randomized trial involving patients undergoing noncardiac surgery, the risk of bleeding was lower with tranexamic acid than with placebo, but noninferiority with respect to cardiovascular complications was not established.
- N Engl J Med 2022; 386:2052-2053
The number of adults who undergo major noncardiac surgery is increasing worldwide,1 including older patients with more coexisting conditions and increased risks of bleeding and thrombotic events.2 Although perioperative bleeding is an important complication that leads to excess morbidity and mortality,3 few therapies have...
- N Engl J Med 2022; 386:1560-1570
A 41-year-old woman with transient dysarthria and facial droop was evaluated for mitral valve masses. Three weeks earlier, she had heavy vaginal bleeding with crampy abdominal pain, fatigue, and lightheadedness. CT imaging was notable for small chronic cerebral infarcts and uterine fibroids. Diagnostic test results were received, and management decisions...
- N Engl J Med 2022; 386:e15
An 18-year-old man with hemophilia A presented with throat discomfort and a recent neck hematoma. The physical examination showed an anterior neck ecchymosis and multiple pharyngolaryngeal hematomas on laryngoscopy. The patient was treated with recombinant factor VIII, and his symptoms and hematomas resolved.
- N Engl J Med 2022; 386:95-95
Forty persons in whom thrombosis and thrombocytopenia had developed after an initial ChAdOx1 nCoV-19 vaccination were given a second dose of a Covid-19 vaccine, most commonly BNT162b2. None of the patients had a recurrence of thrombosis and thrombocytopenia.
- N Engl J Med 2021; 385:2161-2172
A phase 2 trial compared various doses of milvexian with standard enoxaparin for thromboprophylaxis after total knee replacement. Venous thromboembolism occurred in 7 to 8% of patients who received 200 mg of milvexian once or twice daily and in 21% of those who received enoxaparin. The risk of bleeding was...
- N Engl J Med 2021; 385:1961-1973
At a median follow-up of 3.5 years after gene therapy with a factor VIII–containing adeno-associated virus, 16 of 18 men with hemophilia A had enough factor VIII expression that bleeding events were reduced to nearly zero and prophylaxis was discontinued. Vector-related hepatitis led to elimination of the vector in 2...
- N Engl J Med 2021; 385:1815-1816
Platelet-activating antibodies can develop after adenoviral vector vaccination against Covid-19, leading to a potentially fatal procoagulant state. In an analysis involving 35 patients with these antibodies, platelet activation declined in nearly every patient within 12 weeks, despite the persistence of positivity on ELISA, and 5 patients received additional vaccination without...
- N Engl J Med 2021; 385:1680-1689
The phenotypes of VITT were defined in 220 patients in the United Kingdom who presented a median of 14 days after the first ChAdOx1 nCoV-19 vaccination. Half had cerebral venous sinus thrombosis, a third of whom also had intracranial hemorrhage. Mortality was 22%. Intravenous immune globulin may reverse VITT.
- N Engl J Med 2021; 385:1334-1336
Vaccine-induced immune thrombotic thrombocytopenia is caused by the induction of anti-PF4 polyanion antibodies in response to the vaccine. A survey of patients with anti-PF4 antibodies showed no cross-recognition of the SARS-CoV-2 spike protein, and the antiviral antibody levels in response to the vaccine were not correlated with the development of...
- N Engl J Med 2021; 385:885-895
In a multicenter, open-label, randomized trial in patients with immune thrombocytopenia, 91.5% of patients who received mycophenolate mofetil plus glucocorticoids had platelet levels above 100,000 per microliter, as compared with 63.9% of patients who received glucocorticoids only. Quality-of-life measures of physical function and fatigue were worse in the mycophenolate mofetil...
- N Engl J Med 2021; 385:948-950
Idiopathic thrombocytopenia (ITP) has a long history. In 1025, Avicenna described in the Canon of Medicine a patient with characteristics of ITP, which became known as “idiopathic thrombocytopenic purpura.” Splenectomy was the first effective treatment, beginning in 1916, achieving a complete response without recurrence in most patients.1
- N Engl J Med 2021; 385:826-832
Heparin resistance is suspected when more heparin than usual is needed to achieve a therapeutic range of activated partial-thromboplastin time. Management may include increasing the heparin dose, supplementing antithrombin III levels, and using direct thrombin inhibitors.
- N Engl J Med 2021; 385:857-859
Three patients with severe manifestations of VITT after Covid-19 vaccination had a poor response to initial therapy with anticoagulants and intravenous immune globulin. Their conditions improved after therapeutic plasma exchange was initiated, and they were able to leave the hospital.
- N Engl J Med 2021; 385:720-728
A rare side effect of ChAdOx1 nCoV-19 vaccination against Covid-19 — venous or arterial thrombosis, which has been termed vaccine-induced immune thrombotic thrombocytopenia — has been reported in recent months. In this Brief Report, investigators describe the use of intravenous immune globulin in the treatment of three such vaccine recipients...
- N Engl J Med 2021; 385:609-617
In patients undergoing total knee replacement, a single injection of abelacimab (an antibody to factor XI) administered postoperatively was superior to standard care with enoxaparin for the prevention of venous thromboembolism. Bleeding events were rare.