PubMed · 2779888
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1989. Two-dose measles immunization recommendations.. https://pubmed.ncbi.nlm.nih.gov/2779888/
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UNLABELLED: The incidence of prematurity in Germany is about 10% and premature infants with haemophilia A (OMIM 306700) are in fact very rare. We report two new cases, one born in the 28th gestational week, weighing 1200 g with a factor VIII (FVIII) level of 0.03 IU/ml treated with bolus injections of plasma derived FVIII concentrate (pdFVIII), and one born at week 30, weighing only 710 g with a factor level of <0.01 IU/ml and treated with recombinant FVIII concentrate (rFVIII). Recovery of FVIII was 96% in case 1 and 120% in case 2, FVIII half-life was 6 h and 8 h, respectively. During FVIII substitution, neither bleeding, thrombosis nor inhibitor development were noted in both infants. CONCLUSION: Immediate and frequent factor VIII substitution appears to be safe and effective for prophylaxis and treatment in premature haemophilic neonates.
Pompe disease is an autosomal recessive muscle-wasting disorder caused by the deficiency of the lysosomal enzyme acid alpha-glucosidase. Due to virtual absence of acid alpha-glucosidase, patients with classical infantile Pompe disease develop progressive cardiomyopathy, skeletal muscle weakness and respiratory insufficiency leading to death in early infancy. We report on the results of a phase II clinical trial including two patients with classical infantile Pompe disease receiving enzyme replacement therapy over a period of 48 weeks by weekly infusions. Recombinant acid alpha-glucosidase was derived from the milk of transgenic rabbits. Safety was evaluated by recording adverse events while clinical efficacy was evaluated by ventilator-free survival, left ventricular mass index, motor development as well as histologic and biochemical analysis of muscle biopsies. This therapy was in general well-tolerated. There was an overall improvement in left ventricular mass, cardiac function, skeletal muscle function and histological appearance of skeletal muscle.
OBJECTIVE: To investigate the effects of hormone therapy (HT) administered to postmenopausal women on pulmonary function tests (PFT). METHODS: Eighty-two postmenopausal women who were having natural or surgically initiated menopause and had no risk factor that could affect the respiratory system were included into this prospective, randomized study. Twenty-five women who refused to use HT were assessed as the control group (Group I). Nineteen women who accepted using HT and who were having surgically initiated menopause were given continuous estrogen (Group II), 23 were given continuous estrogen and progesterone in combination (Group III) and 15 were given cyclic estrogen and progesterone combination (Group IV). Forced expiratory volume (FEV1), forced vital capacity (FVC), FEV1/FVC, forced expiratory flow rate over 25-75% of the forced vital capacity volume and peak expiratory flow rate were assessed at the beginning of the treatment and in the third month in order to evaluate the effects of HT regimens on the women's PFT. RESULTS: A statistically significant increase was observed only in the FEV1 and FVC parameters of Group III after three months of therapy (P<0.05). The comparison between pre- and posttherapy FEV1 and FVC values showed an increase in the Group IV, but the difference was not statistically significant, while there was no difference between basal and third month FEV1 and FVC values of the group receiving estrogen only. CONCLUSIONS: It was seen that particularly continuous combined HT regimen positively affected the FEV1 and FVC parameters of the postmenopausal women.