Severe cardiac involvement in children with systemic sclerosis and myositis.
OBJECTIVE
To assess the outcome of children with systemic sclerosis (SSc) and features of polymyositis.


METHODS
The charts of 4 children who met the American College of Rheumatology criteria for SSc and had features of polymyositis, as defined by the presence of proximal muscle weakness and elevated serum creatine phosphokinase or aldolase level, were retrospectively reviewed.


RESULTS
All children had multivisceral involvement including (1) myocardial perfusion defects in all cases, with mild to severe dilated cardiomyopathy in 3; (2) lung restrictive syndrome in 3; (3) mild to severe esophageal involvement in all cases; and (4) severe intestinal dysfunction in one child. Combination therapy of corticosteroids, methotrexate (MTX), and cyclosporine resulted in improved skin thickness and muscle strength scores in all cases, as well as in lung restrictive syndrome in 2, but was not effective regarding the progression of intestinal malabsorption in one patient, esophageal dysmotility in 3 patients, and dilated cardiomyopathy in 3. Endstage cardiac failure caused 2 deaths. In one child, heart transplantation was performed for the first time in this indication.


CONCLUSIONS
Children with diffuse cutaneous SSc and features of polymyositis are prone to develop severe cardiomyopathy. Combination therapy of corticosteroids, MTX, and cyclosporine seems to be active on muscle, skin, and lung involvement but does not impair progression of esophageal or myocardial dysfunction. Heart transplantation might be considered, as an experimental treatment, in young patients with severe cardiomyopathy and no other irreversible organ damage.
Quartier
Pierre
P
Unité d'Immunologie-hématologie et Rhumatologie Pédiatrique, Hĵpital Necker-Enfants Malades, Paris, France.
Bonnet
Damien
D
Fournet
Jean-Christophe
JC
Bodemer
Christine
C
Acar
Philippe
P
Ouachée-Chardin
Marie
M
Le Bidois
Jérome
J
Prieur
Anne-Marie
AM
D000293:Adolescent; D009202:Cardiomyopathies / Q000209:etiology* / Q000401:mortality / Q000473:pathology; D002648:Child; D016572:Cyclosporine / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D017285:Polymyositis / Q000150:complications* / Q000188:drug therapy / Q000473:pathology; D011241:Prednisone / Q000627:therapeutic use; D012595:Scleroderma, Systemic / Q000150:complications* / Q000188:drug therapy / Q000473:pathology; D015996:Survival Rate; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D016572:Cyclosporine; D011241:Prednisone; D008727:Methotrexate