Treatment strategies for patients with severe aplastic anemia

Bacigalupo, Andrea

doi:10.1038/bmt.2008.113

Cited by 24 publications

(15 citation statements)

References 23 publications

(19 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…10 Due to increased rates of secondary solid-organ cancer in patients with severe aplastic anemia who received an irradiation-based conditioning regimen, we decided some years ago to use the combination of CY and ATG. Although several studies have confirmed that HSCT after conditioning with CY plus ATG is associated with excellent OS rates, 3,11,12 the long-term follow up of those patients is still limited, with the exception of the Seattle cohort. 6,13 Long-term survivors after HSCT are exposed to late complications including abnormal immune reconstitution, non-malignant organ or tissue dysfunction, delayed infections and also secondary cancers.…”

Section: Introductionmentioning

confidence: 99%

“…Response rate after IST ranges from 57 to 77% at six months and 5-year overall survival (OS) ranges from 60 to 90%. 3,4 However, relapse occurs in one-third of cases 3 and late events such as secondary malignancies or clonal evolution occur in 10%. 5 Hematopoietic stem cell transplantation (HSCT) from an HLA-identical related donor is the treatment of choice for young patients with severe aplastic anemia (SAA).…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Long-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning

Konopacki¹,

Porcher²,

Robin³

et al. 2011

Haematologica

View full text Add to dashboard Cite

The online version of this article has a Supplementary Appendix. BackgroundDue to increased rates of secondary solid organ cancer in patients with severe aplastic anemia who received an irradiation-based conditioning regimen, we decided some years ago to use the combination of cyclophosphamide and antithymocyte globulin. We report the long-term follow up of patients who underwent hematopoietic stem cell transplantation from an HLAmatched sibling donor after this conditioning regimen. Design and MethodsWe analyzed 61 consecutive patients transplanted from June 1991 to February 2010, following conditioning with cyclophosphamide (200 mg/kg) and antithymocyte globulin (2.5 mg/kg/day x 5 days). ResultsMedian age was 21 years (range 4-43); 41 of the 61 patients were adults. Median duration of the disease before hematopoietic stem cell transplantation was 93 days. All but 2 patients received bone marrow as the source of stem cells and all but 2 engrafted. Cumulative incidence of acute grade II-IV graft-versus-host disease was 23% (95%CI 13-34) and 18 developed chronic graft-versus-host disease (cumulative incidence 32% at 72 months, 95% CI 20-46). In multivariate analysis, a higher number of infused CD3 cells was associated with an increased risk of developing chronic graft-versus-host disease (P=0.017). With a median follow up of 73 months (range 8-233), the estimated 6-year overall survival was 87% (95% CI 78-97). At 72 months, the cumulative incidence of avascular necrosis was 21% and 12 patients presented with endocrine dysfunction (cumulative incidence of 19%). Only one patient developed a secondary malignancy (Hodgkin's lymphoma) during follow up. ConclusionsCyclophosphamide and antithymocyte globulin is an effective conditioning regimen for patients with severe aplastic anemia and is associated with low treatment-related mortality. Long-term complications include avascular necrosis and endocrine dysfunction.Key words: hematopoietic stem cell transplantation, severe aplastic anemia, long-term outcomes. Haematologica 2012;97(5):710-716. doi:10.3324/haematol.2011 This is an open-access paper. Citation: Konopacki J, Porcher R, Robin M, Bieri S, Cayuela J-M, Larghero J, XhaardLong-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning

show abstract

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Long-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning

Konopacki¹,

Porcher²,

Robin³

et al. 2011

Haematologica

View full text Add to dashboard Cite

show abstract

“…While some support this treatment as first-line therapy for patients up to the age of 50-55 years, 3,5,6 others limit transplantation as first-line treatment to those younger than 40 years. 1,4,[7][8][9] At these centers, patients aged over 40 years receive immunosuppressive therapy as first-line treatment and transplantation from a matched sibling is reserved to those in whom the immunosuppressive therapy fails.…”

Section: Introductionmentioning

confidence: 99%

Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors

Gupta¹,

Eapen²,

Brazauskas³

et al. 2010

Haematologica

140

108

View full text Add to dashboard Cite

BackgroundTransplantation from an HLA-matched sibling is the treatment of choice for young patients with acquired severe aplastic anemia. For older patients, the acceptable upper age limit for transplantation as first-line treatment varies. The current analysis, therefore, sought to identify age or ages at transplantation at which survival differed. Design and MethodsWe studied the effect of patients' age, adjusting for other significant factors affecting outcomes, in 1307 patients with severe aplastic anemia after HLA-matched sibling transplantation using logistic and Cox regression analysis. Age categories (<20 years, 20-40 years, >40 years) were determined using Martingale residual plots for overall survival and categories based on differences in survival. ResultsPatients aged over 40 years old were more likely to have had immunosuppressive therapy, a poor performance score and a longer interval between diagnosis and transplantation. Neutrophil recovery was similar in all age groups but patients aged over 40 years had a lower likelihood of platelet recovery compared to patients aged less than 20 years (OR 0.45, P=0.01) but not compared to those aged 20-40 years (OR 0.60, P=0.10). Compared to the risk of mortality in patients aged less than 20 years, mortality risks were higher in patients over 40 years old (RR 2.70, P<0.0001) and in those aged 20-40 years (RR 1.69, P<0.0001). The mortality risk was also higher in patients aged over 40 years than in those 20-40 years old (RR 1.60, P=0.008). ConclusionsMortality risks increased with age. Risks were also higher in patients with a poor performance score and when the interval between diagnosis and transplantation was longer than 3 months, implying earlier referral would be appropriate when this treatment option is being considered.Key words: severe aplastic anemia, transplantation, patient age, HLA-identical sibling donor, overall survival. HLA-matched sibling donors. Haematologica 2010;95(12):2119-2125. doi:10.3324/haematol.2010 This is an open-access paper. © F e r r a t a S t o r t i F o u n d a t i o n Citation: Gupta V, Eapen M, Brazauskas R, Carreras J, Aljurf M, Gale RP, Hale GA, OIlhan O, Passweg JR, Ringdén O, Sabloff M, Schrezenmeier H, Socié G, and Marsh JCW. Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors

show abstract

“…Numerous strategies have been applied in the treatment of AA including immunosuppression and/or hematopoietic stem-cell transplantation [8]. Immunosuppression has been found effective in producing complete or partial remission in aplastic anemia.…”

Section: Introductionmentioning

confidence: 99%

Therapeutic Effects of Hydrogen-Rich Solution on Aplastic Anemia in Vivo

Zhao

Mei

Qian

et al. 2013

Cell Physiol Biochem

View full text Add to dashboard Cite

Background: Aplasitc anemia (AA) is a bone marrow failure syndrome characterized by an immune-mediated destruction of hematopoietic stem cells. Though clinical symptoms could be ameliorated by bone marrow transplantation and/or immunosuppressive therapy, frequent recurrence and especially evolution of clonal hematologic diseases remains problematic clinically. Cytokines such as interferon-γ (INF-γ), tumor necrosis factor-α (TNF-α) and interleukin-6 (IL-6) secreted by autologous T cells are closely related with the development of AA. Hydrogen-rich solution was reported to inhibit the levels of cytokines including INF-γ, TNF-α and IL-6 in vivo in recent studies. This study was to investigate the potential therapeutic effects of hydrogen-rich solution on AA in vivo. Methods: AA model was determined in vivo by mice and body weights of the mice were used as the basic physiological index. Peripheral blood cells were calculated to evaluate the hematologic recovery degree. Bone marrow nucleated cells (BMNCs), tissue histology, as well as CFU-S and CFU-GM forming units were used to evaluate the recovery of bone marrow microenvironment. The ratio of CD4⁺ and CD8⁺ cells were examined along with cytokine levels in serum to determine the efficacy of H₂-rich solution on the affected immunological functions. Results: Body weight and number of peripheral blood cells were significantly improved for mice in the H₂-rich solution treated groups as compared with those with AA. The number of BMNCs and CFUs increased markedly and the bone marrow microenvironment was also improved significantly. The experimental group restrained the cell apoptosis, relieved hyperemia and accelerated tissue repair. The number of CD4⁺ and CD8⁺ cells as well as the ratio of CD4/CD8 increased to normal gradually, while the levels of TNF-α, IFN-γ, and IL-6 in serum decreased after H₂-rich solution treatment. Conclusion: Our study firstly showed that hydrogen-rich solution accelerated the recovery of either hematological or immunological recovery on aplastic anemia mice. This finding suggests hydrogen-rich solution as a potential clinical therapeutic agent for AA.

show abstract

Treatment strategies for patients with severe aplastic anemia

Cited by 24 publications

References 23 publications

Long-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning

Long-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning

Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors

Therapeutic Effects of Hydrogen-Rich Solution on Aplastic Anemia in Vivo

Contact Info

Product

Resources

About