PURPOSE: We present clinical and laboratory data on 18 children from 12 hemophilia treatment centers in the United States, Canada, and Europe with the purpose of disseminating information regarding a recently recognized, potentially life-threatening complication of treatment in very young children with hemophilia B. PATIENTS AND METHODS: Twelve hemophilia centers from the United States, Canada, and Europe provided clinical information and laboratory data concerning 18 children who had severe allergic reactions to infused factor (F) IX in close association with the development of an inhibitor to FIX. Laboratory testing for establishment of the diagnosis of hemophilia B and inhibitor to FIX was done locally at the centers treating these patients. FIX gene analysis was performed at one of six molecular genetics institutes. RESULTS: All 18 children had severe hemophilia B, and in each an inhibitor antibody to FIX developed. The median age at the time of anaphylaxis (or anaphylactoid reaction) was 16 months, and the median number of exposure days to FIX was 11. The FIX inhibitor was detected almost simultaneously with the first occurrence of anaphylaxis in 12 of 18 patients. Maximum inhibitor titers were 4.5-600 Bethesda units (BU), with a median titer of 48 BU. FIX gene analysis, performed in 17 of 18 patients, demonstrated complete deletion of the FIX gene in 10 and major derangements in seven. Immune tolerance induction (ITI) regimens have been attempted in 12 patients, with generally poor responses. Two of the 12 experienced nephrotic syndrome while on ITI. Recombinant FVIIa has been successfully used to treat bleeding episodes in 11 of these children. CONCLUSION: Physicians treating young children with hemophilia B should be aware of the potentially life-threatening complication of anaphylaxis. Children with complete gene deletions or major derangements of the FIX gene appear to be at greater risk. Those identified by genotype as being at greater risk may need to receive their first 10-20 treatments in a medical facility equipped for handling such emergencies. Recombinant FVIIa, although not licensed for use in the United States, appears to be the most suitable treatment option for bleeding episodes in such patients.

Anaphylaxis↗

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[Effect of proteins and urea on the renal concentrating ability after chronic water deprivation].

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Animals↗

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The role of endogenous opioid peptides in physiological and pharmacological reward responses--a survey of present-day knowledge.

Endogenous opioid peptides (EOP) participate in a variety of physiological and pharmacological responses that can be recognized by: increased concentrations of EOP in the cerebrospinal fluid; a combination of euphoria and analgesia; and suppression of the responses by specific opiate antagonists. Application of these responses is exemplified in acupuncture, in self-stimulation via hypothalamic electrodes, and in food and fluid consumption. For example, intake of sweet solutions exerts a biphasic effect: in the beginning pain threshold is elevated, but later on tolerance to morphine-induced analgesia develops. A supply of 2% NaCl also stimulates fluid intake as well as raising the pain threshold. Stress-induced analgesia is due to coupled release of ACTH and beta-endorphin from the pituitary. In man, large variations in pain sensitivity may be related to the level of intrinsic EOP.

Analgesia↗

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