title: 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.
abstract: 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.
  
  
  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.
authors.affiliation: Service de Médecine Interne, Hôpital Charles Nicolle, Rouen, France.
authors.forename: Ygal
authors.initials: Y
authors.lastname: Benhamou
authors.forename: Cyrielle
authors.initials: C
authors.lastname: Assié
authors.forename: Pierre-Yves
authors.initials: PY
authors.lastname: Boelle
authors.forename: Marc
authors.initials: M
authors.lastname: Buffet
authors.forename: Rana
authors.initials: R
authors.lastname: Grillberger
authors.forename: Sandrine
authors.initials: S
authors.lastname: Malot
authors.forename: Alain
authors.initials: A
authors.lastname: Wynckel
authors.forename: Claire
authors.initials: C
authors.lastname: Presne
authors.forename: Gabriel
authors.initials: G
authors.lastname: Choukroun
authors.forename: Pascale
authors.initials: P
authors.lastname: Poullin
authors.forename: François
authors.initials: F
authors.lastname: Provôt
authors.forename: Didier
authors.initials: D
authors.lastname: Gruson
authors.forename: Mohamed
authors.initials: M
authors.lastname: Hamidou
authors.forename: Dominique
authors.initials: D
authors.lastname: Bordessoule
authors.forename: Jacques
authors.initials: J
authors.lastname: Pourrat
authors.forename: Jean-Paul
authors.initials: JP
authors.lastname: Mira
authors.forename: Véronique
authors.initials: V
authors.lastname: Le Guern
authors.forename: Claire
authors.initials: C
authors.lastname: Pouteil-Noble
authors.forename: Cédric
authors.initials: C
authors.lastname: Daubin
authors.forename: Philippe
authors.initials: P
authors.lastname: Vanhille
authors.forename: Eric
authors.initials: E
authors.lastname: Rondeau
authors.forename: Jean-Bernard
authors.initials: JB
authors.lastname: Palcoux
authors.forename: Christiane
authors.initials: C
authors.lastname: Mousson
authors.forename: Cécile
authors.initials: C
authors.lastname: Vigneau
authors.forename: Guy
authors.initials: G
authors.lastname: Bonmarchand
authors.forename: Bertrand
authors.initials: B
authors.lastname: Guidet
authors.forename: Lionel
authors.initials: L
authors.lastname: Galicier
authors.forename: Elie
authors.initials: E
authors.lastname: Azoulay
authors.forename: Hanspeter
authors.initials: H
authors.lastname: Rottensteiner
authors.forename: Agnès
authors.initials: A
authors.lastname: Veyradier
authors.forename: Paul
authors.initials: P
authors.lastname: Coppo
mesh_terms: D051722:ADAM Proteins / Q000172:deficiency*; D000071120:ADAMTS13 Protein; D000328:Adult; D000368:Aged; D015331:Cohort Studies; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D015233:Models, Statistical; D011379:Prognosis; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000209:etiology / Q000401:mortality*; D012372:ROC Curve; D012042:Registries; D015203:Reproducibility of Results
chemical_list: D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human