Effective and safe off-label use of caplacizumab treatment in a middle-aged obese male

Ostuni, Angelo; Tiscia, Giovanni Luca; Battista, Cosima; Favuzzi, Giovanni; Montinaro, Vincenzo; Pronzo, Virginia; Cappucci, Filomena; Fischetti, Lucia; Gesualdo, Loreto

doi:10.1016/j.tracli.2020.11.003

Cited by 2 publications

(1 citation statement)

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“…Our study confirms the heterogeneity of iTTP; indeed, our patients showed a variety of clinical manifestations, although the vast majority of patients show neurological symptoms, as often reported in [ 23 , 24 , 25 ]. We found that factors such as neurological or cardiac damage, as well as a delay in diagnosis, are responsible for a worse prognosis.…”

Section: Discussionsupporting

confidence: 88%

Focus on Key Issues in Immune Thrombotic Thrombocytopenic Purpura: Italian Experience of Six Centers

Tiscia

Sartori

Giuffrida

et al. 2021

JCM

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Immune-mediated thrombotic thrombocytopenic purpura is a rare and challenging hematological disease caused by the antibody anti-ADAMTS13. Though the mortality rate has decreased considerably in recent years, fatalities still remain unacceptable. This study aimed at further adding to the existing knowledge of this medical challenge. We enrolled 89 consecutive patients observed in six Italian centers (from 8 August 2013 to 28 May 2021) with a diagnosis of immune-mediated thrombotic thrombocytopenic purpura. Clinical information and blood parameters were collected for all patients. We describe clinical manifestations and laboratory data, possible risk factors and the therapeutic management of first episodes or relapses. A total of 74 first episodes and 19 relapses (median 3 years (interquartile range (IQR): 2–7)) were recorded. Seventy percent of patients enrolled at the first episode showed neurological signs and/or symptoms. All the patients enrolled at the first episode were treated with plasma exchange (median = 12; IQR: 8–19.5) and methylprednisolone (1 mg/kg/day). Rituximab (375 mg/m2 weekly for four weeks) and caplacizumab were given to 15 (20.2%) and 2 patients (2.6%), respectively. We observed an overall mortality of 5.4% in the follow-up (median 60 months; IQR: 36.0–103.5). All fatalities occurred after a diagnostic delay. Present data point to the importance of the early detection of factors mostly associated with poor outcomes. It is likely that use of caplacizumab could improve the prognosis in those patients.

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Section: Discussionsupporting

confidence: 88%