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Chapter 102. Aplastic Anemia, Myelodysplasia, and Related Bone Marrow Failure Syndromes (Part 9)

Outcomes following both transplant and immunosuppression have improved with time. High doses of cyclophosphamide, without stem cell rescue, have been reported to produce durable hematologic recovery, without relapse or evolution to MDS, but this treatment can produce sustained severe fatal neutropenia and response is often delayed. New immunosuppressive drugs in clinical trial may further improve outcome. Other Therapies The effectiveness of androgens has not been verified in controlled trials, but occasional patients will respond or even demonstrate blood count dependence on continued therapy. For patients with moderate disease or those with severe pancytopenia in whom immunosuppression has failed, a 3–4-month trial. | Chapter 102. Aplastic Anemia Myelodysplasia and Related Bone Marrow Failure Syndromes Part 9 Outcomes following both transplant and immunosuppression have improved with time. High doses of cyclophosphamide without stem cell rescue have been reported to produce durable hematologic recovery without relapse or evolution to MDS but this treatment can produce sustained severe fatal neutropenia and response is often delayed. New immunosuppressive drugs in clinical trial may further improve outcome. Other Therapies The effectiveness of androgens has not been verified in controlled trials but occasional patients will respond or even demonstrate blood count dependence on continued therapy. For patients with moderate disease or those with severe pancytopenia in whom immunosuppression has failed a 3-4-month trial is appropriate. Hematopoietic growth factors granulocyte colony-stimulating factor G-CSF granulocyte-macrophage CSF GM-CSF and interleukin 3 IL-3 are not recommended as initial therapy for severe aplastic anemia and even their role as adjuncts to immunosuppression is not well defined. Some patients may respond to combinations of growth factors after immunosuppression has failed. Supportive Care Meticulous medical attention is required so that the patient may survive to benefit from definitive therapy or having failed treatment to maintain a reasonable existence in the face of pancytopenia. First and most important infection in the presence of severe neutropenia must be aggressively treated by prompt institution of parenteral broad-spectrum antibiotics usually ceftazidime or a combination of an aminoglycoside cephalosporin and semisynthetic penicillin. Therapy is empirical and must not await results of culture although specific foci of infection such as oropharyngeal or anorectal abscesses pneumonia sinusitis and typhlitis necrotizing colitis should be sought on physical examination and with radiographic studies. When indwelling plastic catheters become contaminated .