Why the study?
Does allogeneic bone marrow transplantation improve cardiac involvement in children with mucopolysaccharidoses?
Does allogeneic bone marrow transplantation improve cardiac involvement in children with mucopolysaccharidoses?
Allogeneic bone marrow transplantation appears to improve or halt the progression of cardiac involvement in children with mucopolysaccharidoses.
BMT may stabilize cardiac involvement in pediatric MPS; hypothesis-generating and requires prospective confirmation before practice change.
Echocardiography was performed in 16 children undergoing bone marrow transplantation (BMT) for mucopolysaccharidoses. Cardiac involvement before BMT was detected in seven (44%). One year after BMT (11 patients/five with cardiac involvement), left ventricular restriction resolved in 2/3 patients and hypertrophy in one. In the remainder, at mean follow up of 2.5 years, no progression of preexisting or development of new cardiac involvement was noted. It is concluded that in a significant proportion of patients with mucopolysaccharidoses, cardiac involvement improved after BMT.
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Gatzoulis et al. (1995) studied this question.
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