KUDOS TO OUR RESEARCH DEPARTMENT. Recently, Dr. Sunil Babu and members of the research department were able to open an industry trial that may help save a young mother’s life.

On Wednesday, November 25 (the day before Thanksgiving), Babu and Leslie Edgar, RN, CS, NP, research coordinator, were able to make contact and arrangements with Alexion Pharmaceuticals to begin the regulatory process necessary to offer Eclulizumab, an investigational drug, to a 19-year-old female diagnosed with relapsed atypical hemolytic-uremic syndrome.

Atypical hemolytic-uremic syndrome (aHUS) is a serious, life-threatening condition that causes hemolytic anemia, thrombocytopenia, and kidney failure. The disease is very rare, with only three per million in children younger than 18. The incidence in adults is even rarer. Prognosis is typically poor, with most patients either dying or going into end-stage renal failure within a year of diagnosis. Babu’s patient was already on kidney dialysis and requiring blood and platelet transfusions. She had originally been diagnosed in February 2009 with the birth of her first child, but had recently relapsed and required hospitalization. She had already received standard treatment for the condition in February.

Babu determined that a new monoclonal antibody, Eculizumab, was undergoing trials in adult patients with plasma-resistant aHUS. Contact was made with Alexion Pharmaceuticals, and over the Thanksgiving holiday, regulatory documents were forwarded by Angela Hamman, data management, to Edgar.      Contacts were made over home phones and by e-mail. The Western Institutional Review Board amazingly agreed to review the trial on Monday. The study “SWAT team” flew to Fort Wayne Sunday evening and arrived at our Lutheran Hospital office Monday morning to begin the patient screening process. The south office lab and pharmacy handled the pressure and extra work gracefully and without complaint. Carrie Boots, RN, NP, and Babu juggled their schedules with the help of Deborah Meyer-Vilensky, RN, to ensure that the patient and the study team were satisfied. Treatment began successfully on Tuesday, and the patient is currently doing very well and improving. 

The typical industry study startup time is at least four weeks, and includes the preparation of documents, Institutional Review Board (IRB) approval, and the signing of the contract and budget. Even though we’re still tying up loose ends and trying to work through the expedited process, the research department needs to be commended for going the extra mile to ensure that the patient was given the appropriate treatment to promote the best possible outcome possible.