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.

Ygal Benhamou (1) , Cyrielle Assié (1) , Pierre-Yves Boelle (2, 3) , Marc Buffet (2) , Rana Grillberger (4) , Sandrine Malot (2) , Alain Wynckel (5) , Claire Presne (6) , Gabriel Choukroun (6) , Pascale Poullin (7) , François Provôt (2, 8) , Didier Gruson (9) , Mohamed Hamidou (10) , Dominique Bordessoule (11) , Jacques Pourrat (12) , Jean-Paul Mira (13) , Veronique Le Guern (2, 14) , Claire Pouteil-Noble (15) , Cedric Daubin (16) , Philippe Vanhille (17) , Eric Rondeau (18) , Jean-Bernard Palcoux (19) , Christiane Mousson (20) , Cecile Vigneau (21) , Guy Bonmarchand (22) , Bertrand Guidet (2) , Lionel Galicier (23) , Elie Azoulay (2, 24) , Hanspeter Rottensteiner (4) , Agnes Veyradier (25) , Paul Coppo (2)

1 Service de Médecine Interne [CHU Rouen]
2 CHU Saint-Antoine [AP-HP]
3 ESIM - Epidémiologie des maladies infectieuses et modélisation
4 Baxter Innovations GmbH
5 Service de Néphrologie
6 Service de Néphrologie - Médecine Interne
7 Service d'Hémaphérèse - Médecine Interne
8 Service de néphrologie
9 Service de Réanimation
10 Service de médecine interne [Nantes]
11 Service d'Hématologie clinique et thérapie cellulaire [CHU Limoges]
12 Service de Néphrologie - Immunologie Clinique [Toulouse]
13 Service de Réanimation polyvalente
14 Service de Médecine Interne
15 Service de néphrologie et transplantation
16 Service de réanimation médicale [CHU Caen]
17 Service de médecine interne et néphrologie
18 CHU Tenon [AP-HP]
19 Service de Néphrologie pédiatrique
20 Service de néphrologie (CHU de Dijon)
21 Service de néphrologie [Rennes]
22 Service de Réanimation Médicale [CHU Rouen]
23 Service d'immunologie clinique
24 Service de réanimation médicale
25 Service d'Hématologie Biologique [Béclère]

Background. Acquired thrombotic thrombocytopenic purpura is still associated with a 10-20% death rate. So far, early prognostic factors of death could not be clearly identified. To identify prognostic factors associated with 1-month death in thrombotic thrombocytopenic purpura patients with acquired severe (< 10% of normal activity) ADAMTS13 deficiency.Design and Methods. Prospective cohort of patients included between October, 2000, and August, 2010. A validation cohort of patients was set up from September, 2010 to August, 2011. 281 (analysis cohort) and 66 (validation cohort) consecutive adult thrombotic thrombocytopenic purpura patients with acquired severe ADAMTS13 deficiency. 30-day mortality after treatment initiation according to characteristics at inclusion.Results. Non-survivors (11%) were older (P=10-6) and presented more frequently 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 for determining death were age, severe cerebral involvement and LDH level >=10N. 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 intensify patients with a worse prognosis.

Mots clés: thrombotic thrombocytopenic purpura; ADAMTS13; prognostic factor; plasma exchange; rituximab

Domaines: Hématologie

Ygal Benhamou, Cyrielle Assié, Pierre-Yves Boelle, Marc Buffet, Rana Grillberger, et al.. 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.. Haematologica, 2012, 97 (8), pp.1181-6. ⟨10.3324/haematol.2011.049676⟩. ⟨inserm-00700485⟩