Development and validation of a predictive model for death in acquired severe ADAMTS13 deficiency-associated idiopathic thrombotic thrombocytopenic purpura: the French TMA Reference Center experience.

BACKGROUND: Acquired thrombotic thrombocytopenic purpura is still associated with a 10-20% death rate. It has still not been possible to clearly identify early prognostic factors of death. This study involved thrombotic thrombocytopenic purpura patients with acquired severe (<10% of normal activity) ADAMTS13 deficiency and aimed to identify prognostic factors associated with 30-day death. DESIGN AND METHODS: The study involved a prospective cohort of patients and was carried out between October 2000 and August 2010. A validation cohort of patients was set up from September 2010 to August 2011. Altogether, 281 (analysis cohort) and 66 (validation cohort) consecutive adult thrombotic thrombocytopenic purpura patients with acquired severe ADAMTS13 deficiency were enrolled. The study evaluated 30-day mortality after treatment initiation according to characteristics at inclusion. RESULTS: Non-survivors (11%) were older (P=10(-6)) and more frequently presented arterial hypertension (P=5.10(-4)) and ischemic heart disease (P=0.013). Prognosis was increasingly poor with age (P=0.004). On presentation, cerebral manifestations were more frequent in non-survivors (P=0.018) and serum creatinine level was higher (P=0.008). The most significant independent variables determining death were age, severe cerebral involvement and LDH level 10 N or over. A 3-level risk score for early death was defined and confirmed in the validation cohort using these variables, with higher values corresponding to increased risk of early death. CONCLUSIONS: A risk score for early death was defined in patients with thrombotic thrombocytopenic purpura and validated on an independent cohort. This score should help to stratify early treatment and identify patients with a worse prognosis.

[Giant cell arteritis-related upper/lower limb vasculitis]. / Atteintes artérielles des membres supérieurs et inférieurs au cours de la maladie de Horton.

Upper/lower limb vasculitis has been considered an uncommon manifestation of giant cell arteritis, occurring in 3 to 16% of patients. Upper/lower limb vasculitis is still associated with significant morbidity, leading to limb/toe amputation in 5.6 to 15.8% of patients. Yearly clinical vascular examination should be performed systematically to screen upper/lower limb vasculitis at an early stage in patients with giant cell arteritis. Duplex ultrasound has proved to be a reliable non-invasive imaging method for detecting arterial stenoses of the upper/lower limbs in patients with giant cell arteritis. Patients with giant cell arteritis-related upper/lower limb vasculitis should undergo routine investigations to detect underlying aortic complications, concomitant aortic localizations being encountered in more than 50% of cases. Prednisone is the first-line therapy at an initial dose of 0.7-1 mg/kg daily. Prevention of platelet aggregation with low-dose aspirin is potentially effective in preventing ischemic complications of GCA. The indication of surgical therapy should be based on the severity of giant cell arteritis-related upper/lower limb clinical symptoms to avoid unnecessary morbidity in the course of interventional therapy.

Long-term follow-up of upper and lower extremity vasculitis related to giant cell arteritis: a series of 36 patients.

We conducted this retrospective study to determine the prevalence of giant cell arteritis (GCA) in patients exhibiting nonatherosclerotic upper and/or lower extremity arterial involvement and to evaluate the clinical features and long-term outcome of those patients.From January 1997 to March 2008, 36 consecutive patients in the Department of Internal Medicine at the University of Rouen medical center received a diagnosis of symptomatic upper/lower extremity vasculitis related to GCA. In the 36 patients, upper/lower extremity vasculitis preceded the initial GCA diagnosis in 7 patients (19.4%), it was identified in association with GCA in 13 patients (36.1%), and it developed after the onset of GCA in the remaining 16 patients (44.4%). GCA clinical manifestations were severe resulting in ischemic complications of the extremities in 10 patients (27.8%). GCA-related large-vessel involvement was located in the upper extremity alone in 21 patients (58.3%), the lower extremity alone in 7 patients (19.4%), and both the upper and lower extremities in 8 patients (22.2%).Arterial involvement in GCA patients with upper extremity vasculitis was distributed in the subclavian (55.6%), axillary (47.2%), and brachial (22.2%) arteries. In patients with lower extremity vasculitis, involvement included the internal iliac artery (11.1%), common femoral artery (13.9%), superficial femoral artery (33.3%), deep femoral artery (5.6%), and popliteal and anterior tibial arteries (5.6%). Aortic localizations were common in GCA patients with upper/lower extremity vasculitis (68.9% of cases).All patients were given steroid therapy at a median daily dose of 1 mg/kg initially. Reconstructive study was performed in 10 patients (27.8%): venous bypass graft (n = 6), angioplasty (n = 1), thromboendarteriectomy (n = 2), or thrombectomy (n = 1); 2 other patients with extremity ischemia underwent amputation. The median observation time was 32 months; the outcome of upper/lower extremity vasculitis was disappearance of clinical symptoms (44.4%), improvement of clinical manifestations (44.4%), and deterioration of clinical manifestations (11.1%). At last follow-up, the median daily dose of prednisone was 6 mg. Steroid therapy could be discontinued in 12 patients (33.3%).We found that upper/lower extremity vasculitis is not uncommon in patients with GCA, and may be present in the early acute phase of GCA. Nevertheless, because upper/lower extremity vasculitis occurs during the course of GCA, yearly clinical vascular examinations may be adequate to screen for upper/lower extremity vasculitis at an early stage in GCA patients. Early diagnosis of GCA-related upper/lower extremity vasculitis is crucial, and can result in decreased severe ischemic complications. Because aortic localizations were common, GCA patients with upper/lower extremity vasculitis should undergo routine investigations for underlying life-threatening aortic complications (aortic ectasia/aneurysm). We also suggest that patients exhibiting aortic complications should undergo routine clinical vascular examination to detect upper/lower extremity vasculitis.