Session: 322. Disorders of Coagulation or Fibrinolysis: Poster III
Hematology Disease Topics & Pathways:
Biological, Diseases, Therapies, Genetic Disorders, Biological Processes, Protein Disorders, infusion, pathways
She was a 33-year-old woman with a diagnosis of C-PLGD in childhood, after developing ligneous conjunctivitis before the age of 1, developed uterine ligneous lesions and dysmenorrhea as a teenager. At age 27, a hysteroscopy was attempted and laparoscopy was performed which revealed fibrosis and web-like fibrin-rich adhesions in the uterus and fallopian tubes. She was referred to the Oslo University Hospital, Norway, for evaluation and management of her infertility and eye lesions at the age of 28. She was treated with fresh frozen plasma (FFP) infusions with improvement in eye symptoms and dysmenorrhea. At the age of 29, a vaginoscopy and hysteroscopy were performed which again demonstrated fibrin membranes in the uterus. A repeat evaluation at the age of 30 revealed fibrin membranes in the cervix, fibrin accumulation in the uterus, and tube ostia obstructed. At age 33 she was enrolled in a clinical trial, an open-label phase 2/3 study of human Glu-plasminogen administered intravenously at 6.6 mg/kg every 2-4 days in 15 patients with plasminogen deficiency, registered at www.clinicaltrials.gov as #NCT02690714. She received intravenous plasminogen replacement therapy, starting in October 2016. At baseline, she had ligneous conjunctivitis and gynecologic lesions with regular, but atypical menstrual bleeding, discharging very small amounts of thick, mucus-like material over 1-2 days. Her eye lesions had resolved by week 12 of treatment. At the end of the first menstrual cycle on plasminogen treatment, she discharged a large mucus-like plug from the vagina, and had regular and normal menstrual bleeds after that. Approximately 20 weeks into treatment, she reported being pregnant to the investigator. After discussion with the clinical study sponsor, regulatory agencies and the regional ethical committee, the patient and her physician, it was agreed to continue plasminogen replacement therapy through the pregnancy, delivery and recovery period. After approximately 55 weeks into the study treatment, the patient had an uncomplicated pregnancy and delivered a healthy child with APGAR score of 9. The patient remained on PLG treatment post-partum, and her PLG activity trough levels were maintained until she came off the study when her levels went back to baseline. (Figure 1)
Disclosures: Parker: Liminal BioSciences: Current Employment, Current equity holder in publicly-traded company. Trybul: Liminal BioSciences: Current Employment, Current equity holder in publicly-traded company. Sandset: Liminal BioSciences: Other: Investigator Clinical Trial.
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