Aplastic anaemia: an analysis of 174 patients

Abstract

Summary The authors summarize their experience with 174 patients with aplastic anaemia (AA) with particular reference to course, prognostic factors, conversion to other blood disorders, treatment and outcome. Aplastic anaemia was defined as pancytopenia and a hypocellular marrow at some time during the illness. Seven patients terminated with acute leukaemia, 8 developed haematological features of sideroblastic anaemia and 5 showed a red cell membrane defect commonly found in paroxysmal noctural haemoglobinuria. Complete recovery occurred in only 4 patients; 70 others showed a partial remission with some residual haematological abnormality in peripheral blood. Eighteen of these had spontaneous remission. Remission was brief in 27 patients who died; only 32 patients remained in partial remission. Twenty-five per cent. of the patients with AA run an acute course and die within 6 months of the time of diagnosis. The remainder run a subacute or chronic course, punctuated in some cases by a transient remission, but in others by a conversion to other related haematological disorders. An acute course is suggested by a rapid onset of symptoms, a falling neutrophil count, a haemoglobin level less than 5 g/dl and a very low platelet count. A chronic course is likely in those patients who have a slow onset of symptoms, a stable neutrophil count and a Hb level in excess of 5 g/dl. The authors’ experience shows that the disease runs either of the 2 courses irrespective of the supportive therapy.

Proportion of drug‐related serious rare blood dyscrasias: Estimates from the Berlin Case‐Control Surveillance Study

“…Our estimates of the proportion of drug-related blood dyscrasias are similar to previous reports of the drug-attributable fraction for acute agranulocytosis [2], aplastic anemia [1,5], and TTP/HUS [6]. For ITP and IHA, these data, to our knowledge, provide the first systematic estimates.…”

Aplastic Anemia before Bone Marrow Transplantation and Antilymphocyte Globulin

“…The poor prognosis in severely affected patients was still due to early mortality only in a minority; more significant was patients’ refractoriness to platelet substitution resulting from long-standing irreversibility of aplasia, bleeding being responsible for the majority of deaths. Almost identical results were reported by others [8, 9], suggesting that low blood counts alone do not determine the acute risk, but rather reflect the degree of the underlying damage to the hemopoietic stem cell pool itself, or possibly the underlying pathophysiology in a pathophysiologically heterogeneous, albeit phenotypically similar disease. Further retrospective studies in the following years reported overall median survival times between 20 and 60 months.…”

“…There is no doubt that low counts of at least two of the three lineages affected are sufficient to define the subgroup of severe aplastic anemia with poor prognosis, regardless of the therapy chosen [6, 7, 8, 9, 11, 20, 21]. Within this group, a low absolute reticulocyte count [22]and/or a neutrophil count of less then 200/µl suggest a particularly poor prognosis.…”

“…There are only a few data from the time before IST became available. Seven of 174 and 2 of 76 cases of leukemia after aplastic anemia were observed by Mir and Geary [9]and Gordon-Smith [34], respectively. In our series of 113 patients and a median observation time of 3 years, only 1 patient developed AML.…”

Transient pancytopenia

“…On the other hand, idiopathic aplastic anemia or aplastic anemia related to external factors, such as the hepatitis virus or the exposure to a variety of noncytotoxic drugs is usually a chronic disorder with slow and most often incomplete blood cell reconstitution [7,16]. Within the group of pancytopenias with hypoplastic bone marrow, aplasia after cytostatic therapy or exposure to energy rich irradiation is transient.…”