Myelodysplasia: Decision for Death or Disability

Abstract

Treatment of myelodysplastic patients must avoid neglecting a potentially functional child on the one hand and the perpetuation of rejected, brain-damaged cripples on the other. Immediate, complete assessment for brain mass and function and other system anomalies were documented before treatment of 371 patients, with fully informed consent. Of these, 283 had maximum treatment because their brain mass was estimated to be 60 per cent of normal, or greater, and there were no noteworthy complications. The others were treated symptomatically. Seventy-five per cent of patients of normal intellect but paralyzed at high and 90 to 95 per cent of those paralyzed at low levels survived with maximum treatment. Of the patients treated only symptomatically because of criteria excluding maximum treatment, 10 per cent survived to the age of 2 1/2 years. There was a slightly better survival in a group treated symptomatically (42 per cent survival to teen age) before establishment of selection criteria. Children should be treated when parents knowingly accept the ultimate outcome or when doubt of potential exists. Continued reassessment is necessary to alter individual treatment policy. (N Engl J Med 291:1005–1011, 1974)