RESUMEN
A patient with refractory multiple myeloma received an infusion of CTL019 cells, a cellular therapy consisting of autologous T cells transduced with an anti-CD19 chimeric antigen receptor, after myeloablative chemotherapy (melphalan, 140 mg per square meter of body-surface area) and autologous stem-cell transplantation. Four years earlier, autologous transplantation with a higher melphalan dose (200 mg per square meter) had induced only a partial, transient response. Autologous transplantation followed by treatment with CTL019 cells led to a complete response with no evidence of progression and no measurable serum or urine monoclonal protein at the most recent evaluation, 12 months after treatment. This response was achieved despite the absence of CD19 expression in 99.95% of the patient's neoplastic plasma cells. (Funded by Novartis and others; ClinicalTrials.gov number, NCT02135406.).
Asunto(s)
Antígenos CD19/metabolismo , Mieloma Múltiple/tratamiento farmacológico , Receptores de Antígenos de Linfocitos T/uso terapéutico , Adulto , Médula Ósea/inmunología , Médula Ósea/patología , Femenino , Humanos , Mieloma Múltiple/inmunología , Mieloma Múltiple/patología , Inducción de Remisión , Trasplante AutólogoRESUMEN
OBJECTIVES: The principal aims of this study were to evaluate the extent to which patients completed, understood, and were satisfied with the Health Background Questionnaire for Pain (HBQ-P), a health and pain history questionnaire that includes a modification of the Medical Outcome Study Short Form-36, the Treatment Outcomes in Pain Survey, and to examine the degree to which the questionnaire produced reliable and valid responses. A secondary aim was to determine the length of time for a physician to complete the Clinician Evaluation Form for Pain (CEF-P), a brief questionnaire designed to obtain key elements resulting from clinical assessment and management decisions. METHODS: This cross-sectional study utilized data from consecutive new patients seen from January to December 2001 in Drexel University College of Medicine's Pain Medicine and Comprehensive Rehabilitation Center at Graduate Hospital in Philadelphia, PA. The HBQ-P and an accompanying brief satisfaction inventory were completed at home by the patient prior to the individual's initial office visit. The CEF-P was completed by the physician after seeing the patient. RESULTS: Ten of 11 comparisons of patient responses to similar questions on the HBQ-P showed significant consistency. Of eight comparisons between the CEF-P and HBQ-P, two pain duration comparisons showed moderate agreement and one depression comparison showed significant association. Patients consistently had difficulty in answering six single questions and two question sets. Overall patient satisfaction was high. The mean time for the physician to complete the CEF-P was 90 seconds. CONCLUSIONS: Analyses indicate patient responses to similar HBQ-P questions have sufficient reliability to support the use of the Health Background Questionnaire for clinically related data collection and for outcome evaluation of treatments for chronic and recurring pain. The consistently missed questions on the HBQ-P should be revised.