Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra.

OBJECTIVE: To evaluate the quality of life and long-term follow-up of patients enrolled in the Italian registry of cryopyrin-associated periodic syndromes (CAPS). STUDY DESIGN: Since 2004, 20 patients with CAPS were enrolled in a common registry from different Italian Centers of Pediatric Rheumatology; 14 patients were treated with Anakinra in an open fashion. Both treated and untreated patients were routinely followed according to standard of care. The Child Health Questionnaire (CHQ-PF 50) was used to assess the health-related quality of life. RESULTS: The mean duration of follow-up was 37.5 months. In all treated patients, a complete and persistent control of the inflammatory manifestations was observed with no further progression of the disease. At enrollment in the registry, patients showed a poorer health-related quality of life than healthy children in both physical and the psychosocial summary scores. Treatment was associated with a dramatic and sustained amelioration of a variety of measures of poor quality of life, particularly in those concerning the global health perception, bodily pain-discomfort, and other physical domains. CONCLUSIONS: Long-term IL-1 blockade produces a significant and persistent improvement in the clinical manifestations associated with the disease and on the overall quality of life.

B-cell depletion induces prolonged remission in patients with giant cell hepatitis and autoimmune hemolytic anemia.

BACKGROUND: Giant cell hepatitis with autoimmune hemolytic anemia (GCH-AHA) is a rare and severe immune-mediated disorder. Despite aggressive immunosuppressive treatments, the mortality is high. Prednisone has been effectively employed to achieve remission, but with a risk of relapse, if discontinued, and with severe side effects. A possible causative role of humoral immune response has paved the way to anti CD-20 monoclonal antibody (rituximab; RTX). Nevertheless, data about timing of remission and long-term side effects are sparse. METHODS AND MATHERIALS: We have retrospectively evaluated 3 refractory GCH-AHA patients in whom a prolonged remission has been achieved with RTX. In all patients, response to first and second line therapy was incomplete or transitory and severe steroid side effects occurred. RESULTS: A stable and sustained remission was achieved after multiple doses of RTX allowing withdrawing all the other treatments. No life-threatening infections have been recorded, however two patients developed persistent, paucisymptomatic hypogammaglobulinaemia. The only patient who did not develop hypogammaglobulinemia received IgG replacement during RTX. CONCLUSION: RTX induced complete and long-lasting remission allowing discontinuing all the other immunosuppressive drugs. A persistent, paucisymptomatic hypogammaglobulinaemia has been the unique side effect. Although further studies need to replicate our data, RTX can be considered as an effective and safe therapy for sustained remission in patients with severe refractory GCH-AHA.

Attitudes of children with leukemia toward repeated deep sedations with propofol.

Procedural sedation is generally recommended for children requiring repeated painful diagnostic or therapeutic procedures. A child with leukemia undergoes an average of 20 procedures such as lumbar puncture and bone marrow aspiration through the course of illness. No data are currently available about the psychological impact of repeated sedations on children. The objective of this study was to evaluate the attitudes of patients with leukemia toward repeated deep sedations using propofol. A questionnaire addressing sedation-related distress was given to 30 children with leukemia. Procedure-related distress was evaluated using the Amended Observational Scale of Behavioural Distress. Another questionnaire concerning the same issues was given to an historical group of 39 children who had undergone painful procedures without sedation in previous years. Fear and distress were significantly reduced in the sedation group compared with the historical one. Fear of sedation was reported by 17% of children of this group. Distressed behavior was observed in 27%. In conclusion, sedation-related distress was observed in a subgroup of patients; in these cases, specific strategies could be considered to reduce sedation-related distress.