PB1863 APLASTIC ANEMIA ‐ THE CASUISTRY OF A CENTRAL HOSPITAL
Authors: Luís D. , Roque A. , Ribeiro A. , Rito L. , Cortesão E. , Ribeiro L. 
Published: June 2019

Background:

Aplastic Anemia (AA) is a rare haematological disease associated to significant morbidity and mortality, with the autoimmune destruction of hematopoietic stem cells having a central role in its pathogenesis. The diagnostic criteria for AA includes the presence of bone marrow hypoplasia, with no evidence of fibrosis, associated with haemoglobin (Hb) <10 g / dL, platelets <50G / L or neutrophils (ANC) <1.5G / L. The therapeutic algorithm is based mainly on fitness and age, immunosuppression and transfusion support being the standard treatment and allogeneic transplantation of hematopoietic progenitors (ASCT), the only curative treatment.

Aims:

The purpose of this paper is to describe the AA casuistry of the Haematology Service of a Central Hospital.

Methods:

A retrospective study of all patients diagnosed with AA was carried out over a period of 10 years (2008–2018), from the Hematology Service of the Centro Hospitalar e Universitário de Coimbra, in Portugal.

Results:

In the selected period, 34 patients were diagnosed, 71% being female, and the average age at diagnosis was 60 years (18‐85 years). Anemia (44%), haemorrhagic dyskinesia (38%) and infection (3%) were the most frequent reasons for consultation; 15% of the patients were asymptomatic. The lowest values observed were Hb 3.8 g / dL, 0.02G / L ANC and 2G / L platelets. According to Camitta criteria, 17% of patients had non‐severe AAA, 43% severe and 26% very severe.

Of the patients considered fit and considered eligible for ASCH (7 patients ‐ 21%), only 1 had a related donor for which he underwent allogeneic transplantation. Of the remaining patients, 22 received immunosuppressive therapy [9 cyclosporin (CsA) and 13 anti‐thymocyte globulin (ATG) + CsA] and 5 of them received supportive therapy alone. From the statistical analysis performed, TRG showed a favorable trend to CsA + ATG (64%), when compared to CsA and TS (40%) (p = NS), being lower in patients aged ≥ 65 years (27% vs 68%; p = 0.036).

The time to partial response (PR) was higher in the group that did CsA compared to CsA + ATG (15.6 vs 7.8 M; p = 0.016).Complete response (CR) was achieved in 18% and 36% of patients treated with CsA and ATG + CSA (p = NS), respectively. The average overall survival (OS) was 87 months, being lower in the elderly (patients ≥ 65 years, p = 0.024). Despite the absence of significant difference (p = NS), the CsA and CsA + ATG groups showed a tendency to higher OS at 3 M (76% vs 86%, respectively) and 2A (55% vs 64%, respectively), when compared to the group of supportive therapy.

In the 15 patients who died (48.4%), infection was the main cause of death (60%), followed by hemorrhage (20%) and heart disease (20%). The infection was higher in the CsA + ATG group (75% of the causes of death), and was associated with a higher risk of death (p = 0.021).

Summary/Conclusion:

AA is a disease of complex pathophysiology, presenting diagnostic and therapeutic gaps. In the last decade, significant progress has been made to understand its pathophysiology and to find appropriate therapeutic strategies for these patients. In the cohort of patients presented, the analysed variables, namely the rates of response to the therapies and OS, are overlapping with those published in previous studies.

The enlargement of the sample may contribute to a greater robustness of these data, helping in the selection of an individualized therapy.