Pediatr Cardiol 16:61-68, 1995 Pediatric Cardiology 9 Springer-Verlag New York Inc. 1995 Favorable Effects of Immunosuppressive Therapy in Children with Dilated Cardiomyopathy and Active Myocarditis P.R. Camargo.~ R. Snitcowsky, P.L. da Luz, R. Mazzieri, M.L. Higuehi, M. Rati, N. Stolf, M. Ebaid, F. Pileggi Instituto do Cora~ao, Divisao Clfnica, Av. Dr. En~as Carvalho Aguiar 44, Sao Paulo SP 05403-000, Brazil Abstract. Among 68 children with severe dilated cardiomyopathy, 43 (aged 10 months to 15 years) presented with active myocarditis, diagnosed by en- domyocardial biopsy. They were divided into four treatment groups: I, controls: 9 patients submitted to conventional treatment (digitalis, diuretics, and vasodilators) for 8.1 +- 0.7 (SD) months; II, pred- nisone: 12 patients received conventional therapy plus prednisone; III, azathioprine: 16 patients sub- mitted to conventional therapy plus prednisone and azathioprine; ]LV, cyclosporine: 13 patients treated with conventional therapy plus prednisone and cy- closporine. Immunosuppressive therapy was main- tained for a mean of 8.4 _+ 1.2 months. They were submitted to noninvasive (electrocardiogram, chest radiograph, Doppler echocardiogram, and radioiso- topic scintigraphy) and invasive (hemodynamic) studies. In the control group only 2 of 9 patients showed clinical and hemodynamic improvement and 1 of 4, histologic regression of the myocarditis. Among patients submitted to conventional therapy plus prednisone, 3 of 12 presented clinical and he- modynamic improvement; 2 of 5 also showed his- tologic regression of inflammatory process. By con- trast, patients treated with azathioprine or cyclo- sporine associated with prednisone had significantly better results: 13 of 16 and 10 of 13 patients, respectively, had clinical and hemody- namic improvement; all 6 patients in the azathio- prine group and all 4 patients in the cyclosporine group had histologic regression of the myocarditis. Two patients in the prednisone group, one in the azathioprine group, and one in the cyclosporine group died during treatment, in cardiogenic shock. In our experience immunosuppressive therapy with Correspondence to: P.R. Camargo azathioprine or cyclosporine associated with pred- nisone improves the prognosis of children with ac- tive myocarditis and severe ventricular dysfunc- tion. Key words: Active myocarditis -- Endomyocardial biopsy -- Immunosuppressive therapy Active myocarditis is encountered in some children with cardiomyopathy who present with dilatation of one or both ventricular cavities and impaired sys- tolic function. The incidence of active myocarditis varies from 0 to 63% in adult patients with dilated cardiomyopathy [3, 8, 12, 23-25, 28, 32]. To what extent the presence of acute inflammation influ- ences the prognosis is virtually unknown. The avail- ability of an endomyocardial biopsy technique that is safe and easily performed [10, 21, 27, 35] has provided the opportunity to assess the incidence of active myocarditis in patients with dilated cardio- myopathy as well as the effectiveness of therapeutic schemes. Reports [8, 9, 14, 17, 21, 29, 35] of immunosup- pressive therapy in adult patients have offered con- flicting results. Mason et al. [21] reported favorable results in a group of patients with dilated cardiomy- opathy, noting myocardial inflammatory infiltrate on the endomyocardial biopsy specimen. Sekiguchi et al. [31] suggested that active myocarditis remits spontaneously in many patients. Dec et al. [9] have shown that histologic regression or attenuation of myocarditis may occur in the presence of clinical deterioration or vice versa. More recently, Lieber- man et al. [20] found that immunosuppressive ther- apy induced little or no clinical response in patients with myocarditis. Few reports [1, 7, 13, 22] ad- dressed the effects of this therapeutic modality in