ABSTRACT
BACKGROUND: Unproven cellular therapies are being offered to patients for a variety of conditions and diseases for which other treatments have failed. The use of untested cellular therapies is a worldwide problem. Practitioners (e.g., physicians, scientists, QA/QI facility managers, and policy advocates) are perhaps unaware of the risks involved with such therapies. Therefore, a critical need exists to bring attention to the potential limitations and adverse effects of these therapies to inform and limit misinformation. STUDY DESIGN AND METHODS: We describe the extent of the unproven cellular therapy problem through a search of scientific literature and social media coverage. We also describe the regulatory framework that can be used by the practitioner to review and evaluate both proven and unproven cellular therapies. RESULTS: We report on the current state of unproven cellular therapies across the globe. A workflow to facilitate an understanding of the regulatory processes involved in the approval of cellular therapies is provided as well as a list of warnings required by regulatory agencies on various products. It is hoped that this article will serve as a tool kit to educate the practitioner on navigating the field of unproven cellular therapy products. DISCUSSION: Increasing awareness of the issues associated with unproven therapies through education is important to help in reducing misinformation and risks to patients.
Subject(s)
Cell- and Tissue-Based Therapy , Physicians , Cell- and Tissue-Based Therapy/adverse effects , HumansABSTRACT
BACKGROUND: Antibodies to neutrophil-specific antigens are the best characterized cause of transfusion-related acute lung injury (TRALI). CASE REPORT: A double-apheresis platelet (PLT) component was divided and transfused into two patients. One experienced chills, rigors, and dyspnea and the other experienced chills and headache. Transient leukopenia developed in both patients. RESULTS: Evaluation of donor plasma revealed an anti-HNA-2a and no HLA Class I antibodies. The donor had donated 26 previous apheresis PLT components. The 27 donations resulted in 39 separate transfusions and 12 transfusion reactions in 9 patients. Five reactions occurred immediately after the transfusion, 10 within 1 hour, and all within 2.5 hours. Nine of the reactions involved symptoms or signs of pulmonary dysfunction. The symptoms were mild to moderate in nature. None of the inpatients required intensive care transfer nor did any outpatients require hospital admission. Recipient white blood cell (WBC) counts were measured within 8 hours after 38 of 39 transfusions. Leukopenia occurred in 9 of 12 (75%) transfusions with reactions and in 9 of 26 (35%) transfusions without. The reactions did not correlate with pretransfusion WBC count. CONCLUSIONS: Neutrophil antibodies cause a wide variety of transfusion reactions that do not necessarily meet the definition of TRALI. Donors of blood products causing even mild pulmonary reactions or leukopenia should be tested for neutrophil-specific antibodies.