The different results of recent studies of the treatment of rheumatic fever seem to be due not to therapeutic effects but to the inclusion in compared groups of different percentages of patients with inherently different cardiac prognoses. The homogenization of heterogeneous groups has occurred either through insufficient attention to the details of cardiac classification or through inadverent use of different criteria for the classification. The situation has been aptly described by Hill, who wrote, "the essence of a successful controlled clinical trial lies in its minutiae—in a painstaking, and sometimes very dull, attention to every detail." Until the deficiencies in some of these "minutiae" are remedied, many contentions remain unproved.
Massive penicillin treatment, as compared to none, has not demonstrably reduced heart disease. Since massive treatment was not tested against an ordinary streptococcal-eradicating dose of penicillin, the advantages claimed for massive treatment, even if valid, would not pertain to use of the latter regimen. With regard to anti-inflammatory therapy, steroids have been unequivocally superior to salicylates only in temporarily suppressing the acute, overwhelming carditis of a small percentage of patients who are moribund, but even here, steroids have not affected ultimate cardiac status. Such severely ill patients are uncommon. For most patients with rheumatic carditis, the three most satisfactory trials of steroids versus salicylates have indicated a two-to-one vote against any superiority for steroids. In the minority report, the patient population differed significantly from that of the other two reports, and its different results probably arise from this feature. It still remains to be shown convincingly that steroids, salicylates, or both produce more permanent cardiac benefit in most instances than symptomatic therapy alone.
For the practicing clinician, the preceding discussion is intended as a guide for use in reviewing the evidence and making his choice of treatment. For the investigator who performs therapeutic trials, it is intended to indicate the subtle details which are required for a successful study. For both, it should recall the old admonition of an investigative clinician: "Unless prognosis springs from solid diagnostic roots, the tree is unstable."
- Copyright © 1961 by the American Academy of Pediatrics