Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)

Genetics in Medicine

Volumn 18, Issue 1, 2016, Pages 34-40

  McGovern, Margaret M ^a,d   Wasserstein, Melissa P ^a   Kirmse, Brian ^a   Duvall, W Lane ^a   Schiano, Thomas ^a   Thurberg, Beth L ^b   Richards, Susan ^b   Cox, Gerald F ^b,c  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b GENZYME CORPORATION   (United States)

^c BOSTON CHILDREN S HOSPITAL   (United States)

^d STONY BROOK UNIVERSITY   (United States)

Author keywords

acute phase reaction; ceramide; hyperbilirubinemia; Niemann Pick disease type B; recombinant human acid sphingomyelinase

Indexed keywords

ALANINE AMINOTRANSFERASE; ALBUMIN; ALKALINE PHOSPHATASE; ASPARTATE AMINOTRANSFERASE; BILIRUBIN; BIOLOGICAL MARKER; C REACTIVE PROTEIN; CALCITONIN; CERAMIDE; D DIMER; FERRITIN; FIBRINOGEN; GAMMA GLUTAMYLTRANSFERASE; GRANULOCYTE COLONY STIMULATING FACTOR; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; INTERLEUKIN 1ALPHA; INTERLEUKIN 6; INTERLEUKIN 8; IRON; LACTATE DEHYDROGENASE; MACROPHAGE INFLAMMATORY PROTEIN 1ALPHA; MACROPHAGE INHIBITORY PROTEI 1BETA; OLIPUDASE ALFA; PROTEIN; SPHINGOMYELIN; TRANSFERRIN; TUMOR NECROSIS FACTOR ALPHA; TUMOR NECROSIS FACTOR RECEPTOR 2; UNCLASSIFIED DRUG; UNINDEXED DRUG; UROBILINOGEN; IL8 PROTEIN, HUMAN; RECOMBINANT PROTEIN; SPHINGOMYELIN PHOSPHODIESTERASE;

ABDOMINAL PAIN; ACUTE PHASE RESPONSE; ADULT; AREA UNDER THE CURVE; ARTICLE; BACKACHE; BILIRUBIN BLOOD LEVEL; BIOLOGICAL ACTIVITY; BODY MASS; BODY WEIGHT; BRONCHITIS; CELL DEGENERATION; CLINICAL ARTICLE; CONCENTRATION RESPONSE; DIZZINESS; DRUG MEGADOSE; DRUG SAFETY; ENZYME REPLACEMENT; EYE JAUNDICE; FATIGUE; FEMALE; FEVER; GILBERT DISEASE; HEADACHE; HEPATOCYTE DEGENERATION; HUMAN; HYPERBILIRUBINEMIA; HYPERTENSION; IMMUNOASSAY; IRON BLOOD LEVEL; LEG PAIN; LIPOLYSIS; LIVER CELL; LIVER CELL DAMAGE; LIVER DISEASE; LOW DRUG DOSE; MALE; MIDDLE AGED; MYALGIA; NAUSEA AND VOMITING; NEUTROPHIL; NIEMANN PICK DISEASE; ORTHOSTATIC HYPOTENSION; OTALGIA; PARTIAL THROMBOPLASTIN TIME; PHASE 1 CLINICAL TRIAL; PLASMA CONCENTRATION-TIME CURVE; PROTHROMBIN TIME; RETICULOCYTOSIS; SIDE EFFECT; SINGLE DRUG DOSE; SINUSITIS; VOLUME OF DISTRIBUTION; YOUNG ADULT; ADVERSE EFFECTS; CLINICAL TRIAL; DEFICIENCY; DOSE RESPONSE; ENZYMOLOGY; METABOLISM; NIEMANN-PICK DISEASE, TYPE A; NIEMANN-PICK DISEASE, TYPE B; PROCEDURES;

ADULT; C-REACTIVE PROTEIN; DOSE-RESPONSE RELATIONSHIP, DRUG; ENZYME REPLACEMENT THERAPY; FEMALE; HUMANS; HYPERBILIRUBINEMIA; INTERLEUKIN-8; MALE; MIDDLE AGED; NIEMANN-PICK DISEASE, TYPE A; NIEMANN-PICK DISEASE, TYPE B; RECOMBINANT PROTEINS; SPHINGOMYELIN PHOSPHODIESTERASE;

EID: 84954341716     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2015.24     Document Type: Article

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