Long‐term follow‐up of severe aplastic anaemia patients treated with antithymocyte globulin

Abstract

Summary. 468 severe aplastic anaemia (SAA) patients registered in the EBMT-SAA registry who did not undergo bone marrow transplantation and were treated with immunosuppressive therapy (IS; 96% of patients received ATG) were evaluated. Their median age was 23 years (range 1–73) at initial IS therapy, 59% were males; in 69% the aetiology of SAA was idiopathic. Of these 468 patients, 245 had a follow-up of 2 years and 11 are lost. Median follow-up of 223 LTS was 4.1 years (range 2.0–10.9). Comparison of 166 patients who died v. 32%, P9/l (63%v. 80%, P9/l (30%v. 57%. Pv. 79%, Pv. 49%, P0.5 × 10⁹/l, but only after 5 years 80% of cases had platelets > 50 × 10⁹/l. Development of clonal disease was reported of 31 LTS: 19 developed paroxysmal nocturnal haemoglobinuria (PNH), one acute leukaemia, 11 myelodysplastic syndromes and of these 11 five subsequently acute leukaemia. The majority of these patients (23/31) are still alive. Actuarial mortality of LTS is 22% at 8 years, but so far no plateau was achieved. It is concluded that SAA patients who become LTS following IS, show an improvement in haematological status but are probably not cured and are prone to develop clonal (malignant) disease.