Rituximab as first-line therapy for acquired haemophilia A: a single-centre 10-year experience

Haemophilia ◽  
2016 ◽  
Vol 22 (4) ◽  
pp. e338-e341 ◽  
Author(s):  
B. Rossi ◽  
P. Blanche ◽  
V. Roussel-Robert ◽  
A. Berezné ◽  
S. Combe ◽  
...  
HemaSphere ◽  
2019 ◽  
Vol 3 (S1) ◽  
pp. 962-963
Author(s):  
H. Ngu ◽  
D. Simpson ◽  
M. Hanna ◽  
R. Henderson ◽  
E. Merriman ◽  
...  

EP Europace ◽  
2011 ◽  
Vol 13 (5) ◽  
pp. 646-653 ◽  
Author(s):  
H. Tanner ◽  
K. Makowski ◽  
L. Roten ◽  
J. Seiler ◽  
N. Schwick ◽  
...  

1997 ◽  
Vol 78 (06) ◽  
pp. 1463-1467 ◽  
Author(s):  
C R M Hay ◽  
C Negrier ◽  
C A Ludlam

SummaryRecombinant factor Vila was used to treat 38 patients with acquired haemophilia participating in the Novoseven compassionate-use program. 19 were male, median age 59, range 2-89 years. The median pre-treatment anti-human (H) and anti-porcine (P) inhibitor titre was H 43 BU/ml (range 1-4500) and P 4.5 BU/ml (range 0-1600). Recombinant factor VIIa was used as first-line therapy for 14 bleeding episodes and as salvage-therapy for 60 episodes which failed to respond to blood-product therapy given for a median of four days (range 1-21 days) prior to treatment with rVIIa. Pre-rVIIa treatment was not reported for four episodes. The indications for treatment were 7 haemarthroses, 40 muscle haematomas, 20 urinary or GI haemorrhages and 3 surgical interventions. The median starting dose of rVIIa was 90.4 ug/kg (range 45-181). A median of 28 doses (range 1-541) were given per episode, over a median 3.9 days (range 0-43).Efficacy was assessed clinically 8 and 24 h after the start of rVIIa and at the end of treatment. A good response was obtained in all 14 bleeds for which rVIIa was used as first-line therapy. The response after 24 h of rVIIa salvage-therapy for 60 bleeds was good in 75%, partial in 17% and poor in 8%. Efficacy was unreported in 4 cases. The median prothrombin time (PTT) shortened from 12 s (range 9.3-20) pre-treatment to 8.8 s (range 6-14) during treatment. The clinical response did not correlate with the dose of rVIIa used, the type of bleed or the degree of shortening of the PTT following rVIIa infusion.Three patients died from haemorrhagic complications of acquired haemophilia. This mortality of 7.9% is lower than previously reported for this condition. Although one patient developed DIC during treatment with rVIIa, this was probably attributable to hypovolaemic shock, massive transfusion and the use of PCCs. This study demonstrates that rVIIa is a safe, useful and effective treatment for bleeding in patients with acquired haemophilia.


2018 ◽  
Vol 11 (4) ◽  
pp. 1749-1753
Author(s):  
Pietro Ferrara ◽  
Giulia Franceschini ◽  
Lucia Lo Scalzo ◽  
Francesca Ianniello ◽  
Antonio Chiaretti

To assess side effects of MELT monotherapy and MELT in association with oxybutynin in children suffering from nocturnal enuresis (NE). We enrolled 340 enuretic children admitted to our Pediatric Service, Campus Bio-Medico University of Rome, from April 2014 to April 2018; 23 children were excluded. The research was structured in 2 steps. During step 1, a patient’s medical history was carefully collected and physical assessment was performed. During step 2, after 3-month treatment period with MELT (Minirin/dDAVP®) at the dose of 120 mcg a day or MELT plus oxybutynin (Ditropan®), voiding calendar, adherence to treatment and any side effects were examined. The study was conducted in accordance with the Helsinki Declaration. Among 317 patients enrolled in the study, 18 male and 8 female (n=26; 26/317: 8.2%) children, with a mean age 10.86 ± 2.42 years, referred side effects: 13 cases (n=13, 13/26: 50%) treated with MELT monotherapy, 11 cases (n=11, 11/26: 42.3%) treated with MELT plus oxybutynin, 2 cases (n=2, 2/26: 7.7%) who received only oxybutynin. In our research, higher bioavailability of MELT guaranteed lower frequency of adverse effects with a spontaneous and rapid resolution. Several studies demonstrate that dDAVP is an effective and safe drug for NE and MELT formulation is actually considered for first-line therapy of NE, although further research is needed to endorse the observations of the authors.


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