Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000–2011

Abstract

A plastic anemia is a rare life-threatening disease. However, since the introduction of immunosuppressive therapy and allogeneic stem cell transplantation, the outcome has improved considerably, and the 5-year survival is reported to be 70–80% in selected patient cohorts. Yet, contemporary population-based data on incidence and survival are lacking. We performed a national retrospective study to determine the incidence, treatment, and survival of patients with aplastic anemia diagnosed in Sweden from 2000–2011. Patients were included via the National Patient Registry, and diagnosed according to the Camitta criteria. In total, 257 confirmed cases were identified, with an overall incidence of 2.35 (95% CI: 2.06–2.64) cases per million inhabitants per year. Median age was 60 years (range: 2–92), and median follow up was 76 (0–193) months. Primary treatments included immunosuppressive therapy (63%), allogenic stem cell transplantation (10%), or single-agent cyclosporine/no specific therapy (27%). The 5-year survival was 90.7% in patients aged 0–18 years, 90.5% in patients aged 19–39 years, 70.7% in patients aged 40–59 years, and 38.1% in patients aged ≥60 years. Multivariate analysis showed that age (both 40–59 and ≥60 age groups), very severe aplastic anemia and single-agent cyclosporine/no specific therapy were independent risk factors for inferior survival. In conclusion, younger aplastic anemia patients experience a very good long-term survival, while that of patients ≥60 years in particular remains poor. Apparently, the challenge today is to improve the management of older aplastic anemia patients, and prospective studies to address this medical need are warranted.

Revisiting acquired aplastic anaemia: current concepts in diagnosis and management

“…Incidence of aAA varies with age and geographical location. In Europe and North America, the incidence is 2–3 per million per year, and is 2–3‐fold higher in East Asia . The observed sex ratio is close to 1:1.…”

“…Infection is the leading cause of death and patients with PB neutrophils <0.5 × 10 9 /L are at significantly increased risk of severe infection. Duration of neutropenia is a risk factor for infection.…”

Similar outcomes of alemtuzumab-based hematopoietic cell transplantation for SAA patients older or younger than 50 years

“…[1][2][3][4] The retrospective contemporary populationbased study from Sweden highlighted the significantly worse survival among patients with AA aged at least 60 years treated with IST compared with younger patients, with less than 40% alive after 5 years and an excess mortality of 45%. 4,5 Furthermore, after IST and eltrombopag, patients are at later risk for myelodysplastic syndrome/AML and solid tumors, especially lymphomas, in addition to risk for relapse of AA in up to 38% of patients. [42][43][44][45][46] We show for the first time, using a composite end-point of GRFS, that outcomes among older patients with SAA, transplanted mainly from MUDs, were comparable with those for younger patients undergoing FCC transplantation, with low morbidity and lower-than-expected TRM generally observed after IST for SAA in this older age group.…”

“…[1][2][3] For patients aged 50 to 60 years and older than 60 years, 5-year overall survival (OS) is 57% and from 50% to 52%, respectively; response to ATG and CSA among patients older than 40 years and with very severe AA is only 15%. 4,5 In contrast, significant improvement in outcomes after HSCT using matched sibling donor (MSD) and matched unrelated donor (MUD) over time has led to a progressive increase in the upper age limit for consideration of HSCT. [6][7][8] Improvements include better supportive care; improved HLA tissue typing; changes in conditioning regimens including the use of low-dose cyclophosphamide (CY) with fludarabine to replace high-dose CY for MUD HSCT and, in older patients, [9][10][11][12] the use of in vivo T-cell depletion with ATG or alemtuzumab 8,13,14 ; and avoidance of peripheral blood stem cells (PBSC) when using ATG-based conditioning regimens.…”

Pediatric aplastic anemia treatment patterns and responses; power in the numbers

“…The importance of registries focused on AA in particular is reflected in the increasing number of publications describing national outcome data in AA. 2,[12][13][14] In this edition of Haematologica, Rogers et al have made an important contribution to this data pool, informing optimal diagnostic and therapeutic approaches and, equally importantly, highlighting opportunities for further research and discussion in pediatric AA.…”