These therapies have been associated with significant toxicities, and randomized trials demonstrating efficacy are lacking. Recently, defibrotide, a polydeoxyribonucleotide, has been reported to have possible activity as a treatment for veno-occlusive disease. Biopsies reveal some cases to be characterized by diffuse alveolar damage, whereas other cases have a more clearly interstitial component. Treatment with high-dose glucocorticoids is often attempted, but randomized trials evaluating their efficacy have not been performed. Most children will be growth factor deficient and should be treated with replacement therapy.
Ovarian failure develops in most postpubertal women.
Patients treated with high-dose chemoradiotherapy and hematopoietic stem cell transplantation are at increased risk for the development of secondary malignancies. Although complete and sustained engraftment is the general rule after transplantation, in some cases marrow function does not return, and in other cases after temporary engraftment, marrow function is lost. In one large randomized study, previous exposure to chemotherapeutic drugs that are presumed to affect hematopoietic stem cells was highly associated with poor marrow function after transplantation. Graft failure after autologous transplantation can also result from marrow damage during ex vivo treatment, during storage, or after exposure to myelotoxic agents after transplantation. The treatment of graft failure begins with removal of all potentially myelosuppressive agents.