Contributions
Abstract: EP597
Type: E-Poster Presentation
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Pure red cell aplasia (PRCA) is a rare condition characterised by absent erythropoiesis within the bone marrow. The acquired form may present as a primary haematological disorder in the absence of other diseases and is classified as idiopathic PRCA. Occasionally, it can present secondary to other conditions and can be associated with various medications, viral infections, immune disorders, ABO-incompatible haematopoietic stem cell transplantation (HSCT) and malignancies such as thymomas.
Aims
This is a retrospective audit of all adult patients diagnosed with PRCA from 2010 to 2020 to evaluate underlying causes, treatment administered, time to response and survival outcomes.
Methods
A retrospective review of all the patients diagnosed with PRCA above the age of 18 at NUH from 2010 to 2020 was performed. Clinical data recorded include patient’s age at diagnosis, gender, laboratory values at diagnosis, associated conditions, treatment administered, time to response and survival outcomes.
Results
A total of 31 patients were diagnosed with PRCA from our clinical database. There were 20 male and 11 female patients identified. Median age at diagnosis was 58 years (range: 27 – 83 years old). 11 patients were diagnosed with idiopathic PRCA and the rest were diagnosed with secondary PRCA (64.5%). Secondary PRCA was associated with conditions such as thymomas (n=3), autoimmune disease (n=3), drug-related (n=3), ABO-mismatched HSCT (n=7), haematological malignancies (n=2) and parvovirus-induced (n=1). Median haemoglobin at presentation was 6.1 g/dL (range: 3.3 – 12.3 g/dL) and median absolute reticulocyte count was 4.7 X 109/L (range: 1.7 – 27.8 X 109/L). 22 patients were started on immunosuppressive therapy (IST) and 87.5% achieved a complete response. 7 patients were unevaluable for treatment response due to patient’s demise, lost to follow-up or short duration of follow-up. 1 patient was unable to tolerate IST and another patient subsequently lost response and now has refractory PRCA. 14 patients had sustained response to IST but only 14.3% were able to stop IST successfully without relapse. Amongst 4 patients with PRCA associated with thymomas, 2 were diagnosed post-thymectomy and all required adjuvant IST post-thymectomy for sustained response. A total of 26 patients were assessed for survival outcome. 21 patients were alive and 4 were deceased from underlying conditions such as end-stage renal failure (ESRF), relapsed acute leukaemia and infective complications. The cause of death for 1 patient was unknown but likely related to the underlying metastatic thymic carcinoma.
Conclusion
Given that PRCA is a rare diagnosis, the treatment initiated, associated response rates and long- term follow-up for evaluation of the sustainability of those responses are needed to help haematologists better manage these patients.
Keyword(s): Clinical outcome, Pure red cell aplasia, Treatment
Abstract: EP597
Type: E-Poster Presentation
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Pure red cell aplasia (PRCA) is a rare condition characterised by absent erythropoiesis within the bone marrow. The acquired form may present as a primary haematological disorder in the absence of other diseases and is classified as idiopathic PRCA. Occasionally, it can present secondary to other conditions and can be associated with various medications, viral infections, immune disorders, ABO-incompatible haematopoietic stem cell transplantation (HSCT) and malignancies such as thymomas.
Aims
This is a retrospective audit of all adult patients diagnosed with PRCA from 2010 to 2020 to evaluate underlying causes, treatment administered, time to response and survival outcomes.
Methods
A retrospective review of all the patients diagnosed with PRCA above the age of 18 at NUH from 2010 to 2020 was performed. Clinical data recorded include patient’s age at diagnosis, gender, laboratory values at diagnosis, associated conditions, treatment administered, time to response and survival outcomes.
Results
A total of 31 patients were diagnosed with PRCA from our clinical database. There were 20 male and 11 female patients identified. Median age at diagnosis was 58 years (range: 27 – 83 years old). 11 patients were diagnosed with idiopathic PRCA and the rest were diagnosed with secondary PRCA (64.5%). Secondary PRCA was associated with conditions such as thymomas (n=3), autoimmune disease (n=3), drug-related (n=3), ABO-mismatched HSCT (n=7), haematological malignancies (n=2) and parvovirus-induced (n=1). Median haemoglobin at presentation was 6.1 g/dL (range: 3.3 – 12.3 g/dL) and median absolute reticulocyte count was 4.7 X 109/L (range: 1.7 – 27.8 X 109/L). 22 patients were started on immunosuppressive therapy (IST) and 87.5% achieved a complete response. 7 patients were unevaluable for treatment response due to patient’s demise, lost to follow-up or short duration of follow-up. 1 patient was unable to tolerate IST and another patient subsequently lost response and now has refractory PRCA. 14 patients had sustained response to IST but only 14.3% were able to stop IST successfully without relapse. Amongst 4 patients with PRCA associated with thymomas, 2 were diagnosed post-thymectomy and all required adjuvant IST post-thymectomy for sustained response. A total of 26 patients were assessed for survival outcome. 21 patients were alive and 4 were deceased from underlying conditions such as end-stage renal failure (ESRF), relapsed acute leukaemia and infective complications. The cause of death for 1 patient was unknown but likely related to the underlying metastatic thymic carcinoma.
Conclusion
Given that PRCA is a rare diagnosis, the treatment initiated, associated response rates and long- term follow-up for evaluation of the sustainability of those responses are needed to help haematologists better manage these patients.
Keyword(s): Clinical outcome, Pure red cell aplasia, Treatment