A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia

Abstract

One hundred ten patients with severe aplastic anemia were entered into a prospective study. Forty-seven patients who had HIA-identical siblings were treated with marrow transplantation. Sixty-three patients without marrow donors were randomized to supportive care with oral androgen (27 patients). intramuscular androgen (23 patients). or no androgen (1 3 patients). The distributions of the survival times for the patients in the three nontransplantation arms of the study were not different. Transplanted patients had significantly better survival than nontransplanted patients (p 0.0002). Evaluation of prognostic factors for nontransplanted patients showed a better prognosis for patients with unknown etiology than for those with a probable etiology (p = 0.01 ). Also, patients with larger mean red blood cell volumes tended to live longer (p = 0.03). For patients given marrow transplants. significant prognostic fators were age (p 0.01 5) and sex (p 0.027). with better survival for younger male patients. Twenty-seven transplant recipients (57 % ) are alive; 5 have severe chronic graft-versus-host disease, and 22 (47 % ) are living and well. Sixteen nontransplanted patients (25 % ) are alive; 3 continue to have severe disease, and 13 (21 % ) are clinically well and no longer require transfusion support. As used in this study, androgens were of no value in the treatment of patients with severe aplastic anemia. If a suitable donor is available, marrow transplantation is clearly superior to conventional management. at least in younger patients.

High dose intravenous glucocorticoid in the treatment of childhood acquired aplastic anaemia

“…A t least 7 of our patients had a severe form of aplastic anaemia by the criteria of the International Aplastic Anemia Study Group (11). The response rate to the treatment was at least 64 %, including 5 (71%) of those 7 severe cases, 4 of whom (57%) are still alive 2 years later.…”

Clinical impact of HLA‐DR15, a minor population of paroxysmal nocturnal haemoglobinuria‐type cells, and an aplastic anaemia‐associated autoantibody in children with acquired aplastic anaemia

“…Patients with acquired AA were eligible if they met the following criteria: age <18 years, newly diagnosed disease (£180 d) without specific prior treatment, and moderate to very severe AA. Severity was classified as previously described (Camitta et al, 1979;Bacigalupo et al, 1993). The disease was considered severe if at least two of the following were noted: a neutrophil count <0AE5 · 10 9 /l, a platelet count <20 · 10 9 /l, and a reticulocyte count <20 · 10 9 /l with hypocellular bone marrow.…”

Transplantation of highly purified CD34+ progenitor cells from alternative donors in children with refractory severe aplastic anaemia

“…Three were reported previously (Schwinger et al, 2000). The diagnosis of SAA was based on established clinical criteria (Camitta et al, 1979). As all nine patients lacked an HLA-identical family donor and did not respond to one or more courses of standard IST they were selected for this alternative donor (unrelated or mismatched related) transplantation approach.…”

“…Haemopoietic cell transplantation (HCT) is the treatment of choice for younger patients with severe aplastic anaemia (SAA) (Camitta et al, 1979;Storb et al, 1994;Doney et al, 1997;Ball, 2000). If no human leucocyte antigen (HLA)-matched related donor is available, first-line treatment is usually immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporin A (Doney et al, 1997;Fuhrer et al, 1998;Bacigalupo et al, 2000a;Ball, 2000;Kojima et al, 2000;Rosenfeld et al, 2003), the outcome of which is often not optimal.…”