RESUMEN
BACKGROUND: With the recent Food & Drug Administration (FDA) approval of cellular therapy that requires product manipulation prior to administration in combination with a short stability window, the need was identified for local dose preparation within the pharmacy rather than the off-site stem cell processing laboratory. This approval gave rise to assessment of regulatory standards surrounding cellular therapy, evaluation and revision of current standard operating procedures and policies with formal process validation, assessment of occupational exposure mitigation and safety considerations, and development of staff training and education. OBJECTIVE: To describe and provide insight into the stepwise process of FACT validation and onboarding of commercially available cellular therapy products that require sterile compounding manipulation within a pharmacy prior to administration. DISCUSSION: A multidisciplinary effort is required to attain FACT certification and implement pharmacist compounding of cellular therapy products.1 Local preparation within a pharmacy facilitates a sound operational workflow and provides a pathway to perform aseptic manipulations of cellular therapy products safely and efficiently. CONCLUSION: Safe and successful administration of cellular therapies handled and compounded by pharmacy department staff along with program validation requires a preemptive review utilizing a multidisciplinary approach for process development. This manuscript will provide a foundation based on consistency and transparency in effective cellular therapy sterile compounding and aseptic manipulation, proper handling and disposal procedures, increased communication through creation and optimization of treatment plans and order-sets, standardized medical center staff education, and development of policies and standard operating procedures for the entire health care team.
RESUMEN
PURPOSE: Improvements in hemorrhagic and clinical symptoms in a patient with hemorrhagic acute disseminated encephalomyelitis (ADEM) treated with i.v. cyclophosphamide are reported. SUMMARY: A 49-year-old woman was hospitalized with progressively worsening left-sided weakness, dysphagia, diplopia, and vertigo. Shortly before hospital admission, the patient had been treated at another facility with corticosteroids and plasma exchanges. Brain magnetic resonance imaging (MRI) studies conducted at the time of admission showed demyelinating lesions of the pons consistent with ADEM; rapid progression of the patient's symptoms also suggested an autoimmune, demyelinating process, and viral studies ruled out an infectious etiology. Despite initial treatment with i.v. immune globulin and additional corticosteroid courses, the patient's condition continued to deteriorate over the next few weeks, with development of respiratory distress requiring intubation. Repeat MRI revealed a new brain lesion in the splenial region of the corpus callosum, prompting the initiation of i.v. cyclophosphamide therapy (180 mg daily). Approximately 19 days after cyclophosphamide therapy was initiated, an MRI scan revealed substantial reduction of the pontine hemorrhage and a normal splenium appearance, with no new lesion development. The use of cyclophosphamide for hemorrhagic ADEM refractory to other treatments has been previously reported. CONCLUSION: After approximately three weeks of daily i.v. cyclophosphamide therapy, a patient with hemorrhagic ADEM was noted to have stable to improved brain MRI findings along with limited improvement of mental status and movement symptoms.