Treatment of severe aplastic anemia with sequential immunosuppression

Exp Hematol. 1983 Oct;11(9):856-65.

Abstract

Forty-two patients with severe aplastic anemia (SAA) were treated with immunosuppressive therapy (IS) consisting of one or more courses of the following regimens: a) high dose bolus 6 methylprednisolone (BMPr); b) horse antilymphocytic globulin (HALG); c) rabbit ALG (RALG); d) ALG followed by the infusion of haploidentical marrow (ALG +/- BM); e) BMPr in combination with HALG; f) ALG in combination with androgens. All patients received one initial course of IS: 16 (38%) showed hematologic reconstitution and required no further transfusions, of 26 refractory patients 4 died, and 22 received a second course of IS 60 days after the first course. Of these 22, 6 (27%) responded, and of the 16 refractory patients, 9 died, and 7 received a third course of IS 60 days after the second course. Two of these responded (28%), 3 died and 2 are alive but pancytopenic. The overall response rate is 24/42 patients (57%): all of these patients are transfusion independent 6-60 months post-treatment, and 17 are off maintenance therapy with low dose steroids. The actuarial 5 year survival is currently 60%; 13/26 surviving patients have been followed for more than 3 years. The present study confirms that over 50% of patients with SAA can recover from their aplasia following IS treatment, and this is of relevance for patients who can not be offered a bone marrow transplant.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Androgens / administration & dosage
  • Anemia, Aplastic / blood
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Animals
  • Antilymphocyte Serum / administration & dosage
  • Blood Transfusion
  • Bone Marrow Transplantation
  • Child
  • Child, Preschool
  • Clinical Trials as Topic
  • Combined Modality Therapy
  • Female
  • Horses
  • Humans
  • Immunosuppression Therapy* / adverse effects
  • Italy
  • Leukocyte Count
  • Male
  • Methylprednisolone / administration & dosage
  • Middle Aged
  • Platelet Count
  • Prognosis
  • Rabbits
  • Time Factors

Substances

  • Androgens
  • Antilymphocyte Serum
  • Methylprednisolone