Yuval R, Uziel K, Gordon N, Merdler A, Khader N, Karkabi B, Flugelman M Y, Halon D A, Lewis B S
Cardiovascular Clinical Trials Unit, Department of Cardiology, Lady Davis Carmel Medical Center, Haifa, Israel.
Eur J Heart Fail. 2001 Mar;3(2):217-23. doi: 10.1016/s1388-9842(00)00151-3.
Clinical trials, the gold standard for the evaluation of new therapeutic strategies, may prove a drug to be beneficial, harmful or neutral according to its effect on the end-point(s) under study.
To study the reaction and perspective of the patients participating in a clinical heart failure trial, particularly in relation to whether the trial subsequently proved to be positive, negative or neutral.
Anonymous self-completed questionnaire was sent to 78 and returned by 70 consecutive patients 1--6 months after participating in six clinical heart failure trials. The trial was neutral or negative regarding the primary end-point in four (47 patients) of the six studies (MACH-1 trial of mibefradil, REACH trial of bosentan, CASCO trial of calcium sensitizer, ecadotril trial of neutral endopeptidase inhibitor) and positive in two (23 patients) (ICARUS Israel carvedilol study, exercise study of candesartan cilexetil).
Most patients reported subjective global clinical benefit (78% for positive, 74% for negative or neutral trial, NS) after participating in a clinical trial. After adjustment for age, sex, level of education, previous research, perceived comprehension, and treatment allocation (active drug/placebo) in a stepwise regression model, perceived global improvement was greater in older patients (P=0.02), after participation in a positive trial (P=0.05) and in females (P=0.07). The major reason given by the patient for perceived clinical improvement was better follow-up, some believed it was due to change in medication, particularly those who had participated in a positive trial.
More than 70% of patients participating in clinical trials of new drugs for heart failure reported perceived global improvement. Clinical improvement was greater in, but not limited to, patients who participated in positive trials. These salutary findings support the continued recruitment of patients to clinical heart failure trials.
临床试验作为评估新治疗策略的金标准，可根据药物对所研究终点的影响证明其有益、有害或中性。
研究参与临床心力衰竭试验的患者的反应和观点，特别是与该试验随后被证明为阳性、阴性或中性的情况相关。
在连续6项临床心力衰竭试验结束1至6个月后，向78名患者发放匿名自填式问卷，70名患者回复。六项研究中的四项（47名患者）在主要终点方面试验为中性或阴性（米贝拉地尔的MACH-1试验、波生坦的REACH试验、钙敏化剂的CASCO试验、中性内肽酶抑制剂依卡多曲试验），两项（23名患者）为阳性（ICARUS以色列卡维地洛研究、坎地沙坦酯的运动研究）。
大多数患者在参与临床试验后报告有主观整体临床获益（阳性试验为78%，阴性或中性试验为74%，无显著差异）。在逐步回归模型中对年龄、性别、教育水平、既往研究、感知理解和治疗分配（活性药物/安慰剂）进行调整后，老年患者（P = 0.02）、参与阳性试验后（P = 0.05）以及女性患者（P = 0.07）的整体改善感知更强。患者认为临床改善的主要原因是随访更好，一些人认为是药物变化所致，尤其是那些参与阳性试验的患者。
超过70%参与心力衰竭新药临床试验的患者报告有整体改善感知。临床改善在参与阳性试验的患者中更大，但不仅限于此类患者。这些有益发现支持继续招募患者参与临床心力衰竭试验。
