RANDOMIZATION TO SURGERY OR OBSERVATION IN PATIENTS WITH SYMPTOMATIC GALLBLADDER STONE DISEASE - THE PROBLEM OF EVIDENCE-BASED MEDICINE IN CLINICAL-PRACTICE
K. Sondenaa et al., RANDOMIZATION TO SURGERY OR OBSERVATION IN PATIENTS WITH SYMPTOMATIC GALLBLADDER STONE DISEASE - THE PROBLEM OF EVIDENCE-BASED MEDICINE IN CLINICAL-PRACTICE, Scandinavian journal of gastroenterology, 32(6), 1997, pp. 611-616
Background: The description and understanding of gallbladder stone dis
ease in the medical literature are difficult because an assessment of
the natural course of this symptomatic disease, with separation of pat
ients in strictly defined groups, is rally lacking. Method: A multicen
ter study was carried out with patients randomized to either surgery o
r conservative, expectant treatment to examine optimal treatment and n
atural history in well-defined groups of symptomatic gallbladder stone
disease with pain episodes only (study group 1) or acute cholecystiti
s (study group 2). The patients were between 18 and 80 years of age an
d had right upper quadrant or midline epigastric pain and ultrasonogra
phic evidence of gallbladder stone, with or without acute cholecystiti
s. Medical treatment was ordinated on the basis of signs and symptom s
everity. Patients randomized to surgery were placed on the hospital's
waiting list and electively operated on with cholecystectomy as soon a
s conveniently possible. Preliminary results of follow-up are based on
questionnaires mailed al regular intervals and consultations if requi
red by the patients' symptoms. Quality of life and freedom from pain a
re study end points for evaluation. Results: Five hundred and one pati
ents were considered for the study, 318 patients (63.5%) belonging to
group 1 and 183 (36.5%) to group 2. One hundred and eighty-four patien
ts (36.7%) were randomized, 120 (24%) in group 1 and 64 (12.7%) in gro
up 2. Three hundred and seventeen patients (63.3%), with an equal prop
ortion from each study group, were excluded, the largest number of pat
ients because of incapacitating pain or serious presenting symptoms (2
6%). More women (81%) than men (19%) had pain alone (group 1), bur the
sex ratio was reduced, 56% women and 44% men, in patients with acute
cholecystitis (group 2): with a highly significant difference (P = < 0
.001) between the two groups. A significant difference in patient with
drawal from randomized treatment was registered, with 24% from observa
tion and 12% from surgery (P = 0.032), but with an equal distribution
in the two study groups. Conclusions: A randomized trial of this natur
e is feasible but extremely difficult to perform because of the hetero
geneous nature of gallbladder stone disease, leading to exclusion of m
any patients and difficulties in measuring and evaluating outcome vari
ables.