European Association of Hospital Pharmacists (EAHP) guidance on the pharmacy handling of in vivo gene therapy medicinal products.

Gene therapy is becoming increasingly prevalent, with new gene therapy medicinal products (GTMPs) being approved for use every year. Hospital pharmacists are expected to prepare and dispense these products, but there is substantial heterogeneity in the availability of up-to-date, practical guidance at a national level in Europe. Many institutions have no or very limited experience in handling GTMPs.

Implementation and operational management of marketed chimeric antigen receptor T cell (CAR-T Cell) therapy-a guidance by the GoCART Coalition Pharmacist Working Group.

Chimeric Antigen Receptor T cells (CAR-T cells) are a type of Advanced Therapy Medicinal Product (ATMP) classified as ex-vivo (cell-based) gene therapy. CAR-T cells constitute an immunotherapy that works by enabling T cells to specifically recognise cancer cells and destroy them [1]. CAR-T cells are currently licensed to treat certain blood cancers including relapsed or refractory lymphomas, B-cell acute lymphoblastic leukaemia or multiple myeloma [2].

A randomized clinical trial of histamine 2 receptor antagonism in treatment-resistant schizophrenia.

Histamine has important functions as regulator of several other key neurotransmitters. Patients with schizophrenia have lower histamine H1 receptor levels. Since a case report in 1990 of an effect of the H2 antagonist famotidine on negative symptoms in schizophrenia, some open-label trials have been performed, but no randomized controlled trial.

Administration of vitamin K to newborn infants and childhood cancer.

Objectives: To investigate whether childhood cancer is associated with intramuscular administration of vitamin K to newborn infants.

Design: Routines for administration of vitamin K to infants born after normal deliveries during 1973-89 were obtained from maternity hospitals. Occurrence of cancer up to the end of 1991 was identified by comparing these records with the national cancer registry.

Homozygous protein C deficiency in two siblings.

Homozygous protein C (PC) deficiency is reported in two siblings (girl and boy) who received their proper diagnoses at the ages of 7 4/12 and 1 3/12 years respectively. The girl had perinatal asphyxia without bleeding. At 1 year of age she developed purpura fulminans.

Four pairs of siblings with acute leukemia during 1966-1985 in the Nordic countries: indication of an elevated familial risk?

Four pairs of siblings with acute leukemia in the Nordic countries during 1966-1985 are reported. The national data indicate that the risk of leukemia is 5.9 times greater in siblings of children with leukemia.

Combined deficiency of coagulation factors II, VII, IX, and X: a case of probable congenital origin.

Combined deficiency of coagulant activity of the vitamin K-dependent factors was found in a 14-year-old boy suffering from severe hemorrhages. Immunoassays revealed the presence of acarboxyprothrombin. The bleedings could be controlled, but the coagulation defects persisted during more than 2 years' follow-up and could not be corrected by oral or parenteral vitamin K.

Variations of protease inhibitors in foetuses, newborn infants and in some neonatal disorders.

Low levels of protease inhibitors have been found on the 1st day of life in IRDS infants. 19 IRDS infants were studied together with foetuses and control term and preterm infants. Alpha1-antitrypsin, antichymotrypsin and alpha2-macroglobulin were measured with the electroimmuno assay.

Abnormal proteolysis in sick newborns.

87 newborn infants were studied on their first day of life for defects in the coagulation and fibrinolytic systems. The infants were divided into two diagnostic groups, one with IRDS, the other with mixed neonatal disorders. Factor V, fibrinogen and fibrin/fibrinogen degradation products (FDP) were abnormal more often than any of the other factors examined.

Cytarabine treatment of herpes simplex encephalitis in infants and small children. A report on three cases with a short review of the literature.

Three cytarabine-treated infants and children with herpes simplex encephalitis are presented. The effect of the treatment was excellent in 2 cases. One boy who had a CP syndrome died.