Search

Introduction: The clinical benefits of administering low-dose prophylaxis in children with haemophilia are well established. Qualitative research describing the impact of prophylaxis on quality of life is comparatively rare in this area., Aim: The aim of this study was to investigate in children the experiences of living and becoming adjusted to haemophilia before prophylaxis, by collecting information directly from children and their parents or guardians. A further goal was to evaluate whether and how the use of low-dose prophylaxis impacts the disease experience., Methods: A grounded theory design according to Strauss and Corbin was chosen for this study. The study was conducted in the Haemophilia Treatment Centre at Aluva, Kerala, India and involved nineteen participants (children, mothers, father and grandmothers) who were selected by theoretical sampling. Data were collected through audiotaped interviews, which included demographic and semi-structured interview questions. Data were coded and evolved into concepts and categories that lead to the emergence of theory., Results: The study resulted in the construction of 'Theory of Therapeutic Metamorphosis'. It comprised two stages: stage of bondage (enduring hardships), experienced during the absence of prophylaxis or on-demand treatment and stage of freedom (deliverance/reductions, energized life/improvements and behaviour to seek prophylaxis) experienced during low-dose prophylaxis., Conclusion: This study illustrates the challenges faced by children with haemophilia and their families and the positive impact of low-dose prophylaxis. Further prospective research studies are required to add to the growing knowledge in this area., (© 2020 John Wiley & Sons Ltd.)

We report the use of rituximab (MabThera); Roche Grenzach-Wyhlen, Germany) in a 6-year-old boy with severe haemophilia A and a high titre alloimmune factor VIII (FVIII) antibody, which had failed to respond to standard immune tolerance therapy. Rituximab was administered in 4 weekly doses with concurrent high-dose i.v. immunoglobulin (Flebogamma); Grifols, Barcelona, Spain) followed by daily high-dose recombinant FVIII concentrate (Recombinate); Baxter, CA, USA). Despite a fall in CD20 positive cell count to undetectable levels the inhibitor persisted. We discuss the possible reasons for failure of immune tolerance induction and review the literature concerning the use of rituximab for this indication.

Background and Objectives: Recombinant activated factor VII (rFVIIa/NovoSeven) has been advocated in the treatment of life-threatening haemorrhage, but appropriate clinical indications remain uncertain. The aim of this study was to detect factors predictive of outcome and to incorporate them into a prognostically significant scoring system., Materials and Methods: Thirty-six patients received rFVIIa for uncontrolled surgical, traumatic or obstetric bleeding in the Northern Region of the UK over a 45-month period. Clinical, laboratory and outcome data were examined. Characteristics of survivor and non-survivor groups were compared. A prognostic scoring system was evaluated retrospectively according to the presence of coagulopathy, renal impairment, hypothermia, greater than 10 units of red cell transfusion, advanced age and obstetric indication, with patients allocated to low, intermediate and high-risk groups., Results: Clinical response occurred in 26 patients (72%) with a reduction in prothrombin time and blood product requirements. Death occurred in 19 (53%). Four patients (11%) suffered thrombotic events. Survivors were younger than non-survivors and less likely to have coagulopathy, renal impairment or hypothermia at the time of administration. Survivors were more likely to have had an initial clinical response in terms of an immediate reduction in haemorrhage. Non-survivors were transfused a greater number of red cell units prior to administration. Survival varied according to prognostic score; low-risk patients had a survival rate of 85%, intermediate-risk patients had a survival rate of 50% and high-risk patients had a survival rate of 18%., Conclusions: FVIIa has a role in the cessation of haemorrhage, but may not improve survival. Use of a clinical scoring system may help to predict outcome.