Oioioi, fortsetter Ă„ komme drypp fra OMER:
Pasient som ikke kunne behandles med dagens standardlegemiddel Soliris (https://en.wikipedia.org/wiki/Eculizumab) ble reddet av OMS721.
Title: Omeros Announces OMS721 Presentation at Annual Meeting of the European Society for Blood and Marrow Transplantation
Date(s): 28-Mar-2017 7:01 AM
â Additional Positive âChallenge-Rechallengeâ Data Reported in Patient with Stem Cell Transplant-Associated TMA â
SEATTLEâ(BUSINESS WIRE)âMar. 28, 2017-- Omeros Corporation (NASDAQ: OMER) today announced presentation of a case report describing resolution of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) in a dialysis- and transfusion-dependent adolescent girl who was treated with OMS721 under a compassionate-use protocol. The presentation âResolution of acute kidney injury secondary to TA-TMA by the anti-MASP-2 monoclonal antibody OMS721 in a pediatric HSCT recipientâ occurred at the 43rd Annual Meeting of the European Society for Blood and Marrow Transplantation in Marseille, France on Monday, March 27, 2017. Marco Zecca, M.D., Director of Pediatric Oncology at the Fondazione IRCCS Policlinico San Matteo, presented the data. OMS721 is Omerosâ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.
The presentation describes a girl who developed HSCT-TMA following stem cell transplantation at 14 years of age. She was initially treated with eculizumab but did not tolerate treatment, developing pulmonary edema that recurred on retreatment with eculizumab. The HSCT-TMA became life-threatening and she required hemodialysis and daily platelet transfusions. Dr. Zecca, the patientâs physician, requested OMS721 for compassionate-use treatment and Omeros complied. Following OMS721 treatment, the patient was able to discontinue hemodialysis and to decrease substantially her platelet transfusion requirements. Recently, the patientâs dose of OMS721 was tapered, but she developed a viral infection that reactivated her HSCT-TMA. Her TMA was again successfully treated with restoration of the original OMS721 dose. To date, she has remained free of both dialysis and transfusions.
âThis patient had severe TMA that I believe would have caused her death,â stated Dr. Zecca. âHer positive response to OMS721 treatment, both initially and following her virus-induced relapse during tapering, was impressive - the results of OMS721 treatment in this challenge-rechallenge scenario underscore the important effects of the drug. Since the poster was produced, her TMA has remained in remission and we have been able to discontinue her platelet transfusions. Her rapid response has been heartening, and we all are grateful for this remarkable outcome.â
Thrombotic microangiopathy is a potentially life-threatening complication of HSCT. Approximately 20,000 HSCT procedures are performed in the U.S. annually, and TMA is reported to occur in up to 30 percent of HSCT patients. Although the kidney is the most commonly affected organ, HSCT-TMA is a multi-system disorder and can also manifest clinically in the lungs, gastrointestinal tract and central nervous system. Reported mortality in patients with multi-organ involvement is greater than 90%. Even in patients who survive acute episodes, HSCT-TMA increases the risk for chronic kidney disease and end-stage renal disease.
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âI say this with humility, we are saving livesâ sitat deres CEO denne mĂ„neden.