Effective treatment of aplastic anemia: A Synthesis of Findings from 13 Studies

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Original Abstract of the Article

Major Research Findings

Aplastic anemia is a rare and serious disease in which the bone marrow does not produce enough blood cells. This can lead to a variety of symptoms, including fatigue, weakness, shortness of breath, and easy bruising or bleeding. 12 suggests that Anti-thymocyte globulin (ATG) combined with eltrombopag is the standard immunosuppressive treatment for aplastic anemia. ATG plays a critical role in the treatment of severe aplastic anemia. 1 found that allogeneic bone marrow transplantation is an effective treatment for severe aplastic anemia. Patients who underwent transplantation from HLA-identical siblings showed improved survival rates and many returned to normal activities. 7 demonstrated that antithymocyte globulin combined with cyclosporin A is an effective regimen for the treatment of aplastic anemia. This treatment showed a higher response rate than other treatments, particularly in patients with severe aplastic anemia. 4 reported that integrated Chinese and Western medicine is an effective treatment option for aplastic anemia. The treatment using fetal blood transfusion, Chinese medicinal herbs and vitamin C showed higher effectiveness compared to the control group, especially in chronic aplastic anemia. 11 conducted a comparative study between HLA-matched sibling donor hematopoietic stem cell transplantation and first-line immunosuppressive therapy. HLA-matched sibling donor hematopoietic stem cell transplantation is a strong option for aplastic anemia, but for patients without a matched sibling, immunosuppressive therapy can be used as an alternative treatment. 9 evaluated the efficacy of different immunosuppressive therapy regimens for children with aplastic anemia. ATG combined with cyclosporin A was found to be the most effective treatment, showing high response rates and 5-year survival rates. 3 showed that antithymocyte globulin alone is effective in improving hematopoiesis in some patients with aplastic anemia. 5 revealed that antilymphocyte globulin combined with cyclosporine A is more effective than antilymphocyte globulin alone for the treatment of severe aplastic anemia. The combination therapy showed higher response rates, lower risk of early mortality and shorter time to red cell transfusion independence. 6 demonstrated that adding hematopoietic growth factors to antilymphocyte globulin and cyclosporine A improves treatment effectiveness. Adding growth factors to this treatment regimen resulted in higher response rates, lower rates of early infection and mortality, and faster recovery of bone marrow hematopoiesis. 13 reviewed and analyzed data comparing the effectiveness of peripheral blood stem cell transplantation and bone marrow transplantation for aplastic anemia. Although peripheral blood stem cell transplantation is gradually replacing bone marrow transplantation as the standard procedure for treating aplastic anemia, it remains unclear which approach is superior. 8 compared different immunosuppressive regimens and found that the combination of ATG and cyclosporine A was the most effective treatment for patients with aplastic anemia. 2 reported that early bone marrow transplantation can be an effective treatment option for severe aplastic anemia. Early transplantation resulted in faster bone marrow recovery and decreased mortality.

Treatment Summary

The treatment of aplastic anemia varies depending on the severity of the disease and the patient's condition. 12 recommended Anti-thymocyte globulin (ATG) combined with eltrombopag as the standard immunosuppressive treatment for aplastic anemia. 1 presented HLA-matched sibling donor bone marrow transplantation as an effective treatment for severe aplastic anemia. 7 showed that the combination of ATG and cyclosporin A is particularly effective in patients with severe aplastic anemia. 4 presented integrated Chinese and Western medicine as an effective treatment for aplastic anemia. 11 introduced immunosuppressive therapy as an alternative treatment for patients without HLA-matched siblings. 9 highlighted that ATG combined with cyclosporine A is the most effective immunosuppressive therapy for children with aplastic anemia. 3 showed that ATG alone can be an effective treatment option. 5 highlighted that the combination of antilymphocyte globulin and cyclosporine A is more effective than antilymphocyte globulin alone. 6 introduced a treatment regimen involving adding hematopoietic growth factors to antilymphocyte globulin and cyclosporine A. 13 discussed the effectiveness of both peripheral blood stem cell transplantation and bone marrow transplantation, but it remains unclear which is superior. 8 indicated that ATG combined with cyclosporine A is the most effective treatment for aplastic anemia. 2 presented early bone marrow transplantation as an effective treatment option for severe aplastic anemia.

Benefits and Risks

Benefit Summary

Various treatments have been developed for aplastic anemia, and their effectiveness has been demonstrated. 12 suggests that the combination of ATG and eltrombopag is the standard immunosuppressive treatment for aplastic anemia. 1 found that bone marrow transplantation is an effective treatment for severe aplastic anemia and can lead to improved survival and return to normal activities. 7 demonstrated that the combination of ATG and cyclosporine A is particularly effective in patients with severe aplastic anemia. 4 showed that integrated Chinese and Western medicine is more effective than traditional Western medicine, especially for patients with chronic aplastic anemia. 9 found that ATG combined with cyclosporine A is the most effective immunosuppressive therapy for children with aplastic anemia. 2 revealed that early bone marrow transplantation can lead to faster bone marrow recovery and reduced mortality.

Risk Summary

There are various risks associated with aplastic anemia treatments. 1 identified graft-versus-host disease and infections as major risks associated with bone marrow transplantation. 7 mentioned the risk of relapse and development of secondary diseases with the combination of ATG and cyclosporine A. 9 reported the risk of infections with immunosuppressive therapies. 2 reported the risk of infections associated with early bone marrow transplantation.

Comparison of Studies

Commonalities of Studies

Multiple studies have shown that the combination of ATG and cyclosporine A is an effective treatment for aplastic anemia. Bone marrow transplantation has also been identified as an effective treatment option for severe aplastic anemia. 12 , 1 , 7 , 9 , 2

Differences Between Studies

Studies differed in their treatment methods, target patient groups, and evaluation methods. 4 presented integrated Chinese and Western medicine as an effective treatment option. 11 highlighted that immunosuppressive therapy can be an alternative treatment for patients without HLA-matched siblings. 6 introduced a treatment regimen involving adding hematopoietic growth factors. 13 compared the effectiveness of peripheral blood stem cell transplantation and bone marrow transplantation but did not determine which is superior. 8 conducted a comparison of different immunosuppressive therapy regimens.

Consistency and Discrepancies in Results

Multiple studies consistently indicate that the combination of ATG and cyclosporine A is an effective treatment for aplastic anemia. 12 , 7 , 9 , 5 However, there are inconsistencies in the results of studies regarding bone marrow transplantation. 1 found that HLA-matched sibling bone marrow transplantation is an effective treatment for severe aplastic anemia, while 11 suggested that immunosuppressive therapy is an alternative option for patients without HLA-matched siblings. 2 showed that early bone marrow transplantation is a viable treatment option.

Considerations for Real-World Application

The treatment of aplastic anemia is highly individualized and depends on the severity of the disease and the patient's condition. 12 suggests that ATG combined with eltrombopag is the standard immunosuppressive treatment for aplastic anemia. 1 presented HLA-matched sibling donor bone marrow transplantation as an effective treatment for severe aplastic anemia. 7 showed that the combination of ATG and cyclosporine A is particularly effective in patients with severe aplastic anemia. 4 presented integrated Chinese and Western medicine as an effective treatment for aplastic anemia. 11 introduced immunosuppressive therapy as an alternative treatment for patients without HLA-matched siblings. 9 highlighted that ATG combined with cyclosporine A is the most effective immunosuppressive therapy for children with aplastic anemia. 3 showed that ATG alone can be an effective treatment option. 5 highlighted that the combination of antilymphocyte globulin and cyclosporine A is more effective than antilymphocyte globulin alone. 6 introduced a treatment regimen involving adding hematopoietic growth factors to antilymphocyte globulin and cyclosporine A. 13 discussed the effectiveness of both peripheral blood stem cell transplantation and bone marrow transplantation, but it remains unclear which is superior. 8 indicated that ATG combined with cyclosporine A is the most effective treatment for aplastic anemia. 2 presented early bone marrow transplantation as an effective treatment option for severe aplastic anemia. It is crucial to consult with a physician to determine the most appropriate treatment option for each individual based on their specific condition.

Limitations of Current Research

Research into aplastic anemia treatments is still ongoing and there are areas that need further investigation. 12 , 1 , 7 , 4 , 11 , 9 , 3 , 5 , 6 , 13 , 8 , 2 More research is needed to develop more effective treatment options.

Future Research Directions

Future research on aplastic anemia treatments should focus on the following key areas:

  • Development of new treatment options
  • Further validation of the effectiveness of existing treatments
  • Individualized treatment approaches
  • Understanding the underlying mechanisms of the disease

These research efforts will contribute to further advancements in the treatment of aplastic anemia.

Conclusion

Aplastic anemia is a serious disease characterized by the bone marrow's inability to produce enough blood cells. 12 , 1 , 7 , 4 , 11 , 9 , 3 , 5 , 6 , 13 , 8 , 2 While various treatment options have been developed, more research is needed to find a cure for this disease. It is essential to consult with a physician to determine the best treatment approach for each individual based on their specific condition.

Treatment List

Anti-thymocyte globulin (ATG), eltrombopag, cyclosporin A, bone marrow transplantation, peripheral blood stem cell transplantation, integrated Chinese and Western medicine

Literature analysis of 13 papers
Positive Content
13
Neutral Content
0
Negative Content
0
Article Type
9
3
3
1
12
1.

Aplastic anemia treated by allogeneic bone marrow transplantation: a report on 49 new cases from Seattle.

Author: StorbR, ThomasE D, WeidenP L, BucknerC D, CliftR A, FeferA, FernandoL P, GiblettE R, GoodellB W, JohnsonF L, LernerK G, NeimanP E, SandersJ E

Aplastic anemia treatment
GVHD
Graft rejection
Infection

Marrow transplantation was found to be an important treatment for severe aplastic anemia, with 41% of patients surviving long term. Marrow graft rejection, graft-versus-host disease, and infections remain the major causes of failure.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.
Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, U.S. Gov't, P.H.S.Research funding provided by the Public Health Service (PHS).

Language : English

2.

Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality.

Author: CamittaB M, ThomasE D, NathanD G, SantosG, Gordon-SmithE C, GaleR P, RappeportJ M, StorbR

Decreased mortality
Full marrow reconstitution
Graft-versus-host disease

A study of 67 patients with severe aplastic anemia compared early bone marrow transplantation to conventional treatments. Patients who received early transplants (within 33 days of diagnosis) had significantly higher survival rates (24 out of 36 alive after 9 months) and faster recovery of marrow function compared to those receiving conventional treatments (12 out of 31 alive). Early transplantation also significantly reduced the risk of death within the first 3 months. While longer follow-up is needed, early bone marrow transplantation appears to be the most effective treatment for severe aplastic anemia.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, U.S. Gov't, P.H.S.Research funding provided by the Public Health Service (PHS).

Language : English

3.

Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial.

Author: ChamplinR, HoW, GaleR P

Improved hematopoiesis
No complete recovery

This study found that antithymocyte globulin was effective in improving hematopoiesis in some patients with aplastic anemia, with 11 out of 21 patients showing sustained improvement within three months, compared to no improvement in the control group.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Research Support, U.S. Gov't, P.H.S.Research funding provided by the Public Health Service (PHS).

Language : English

4.

[Clinical observation on child aplastic anemia treated with integrated Chinese and Western medicine].

Author: ZhangZ M, ShiB J, FanR

Improved hematopoietic function
Unknown long-term effects

This study found that fetal blood transfusion, Chinese medicinal herbs, and vitamin C were more effective in treating aplastic anemia than Western medicine alone.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : Chinese

5.

[Antilymphocyte globulin combining with cyclosporine A in the treatment of severe aplastic anemia].

Author: ZhengY, ChuY, ShaoZ

Higher response rate
Lower mortality risk

Intensive immunosuppressive therapy (IIST) with antilymphocyte globulin (ALG) and cyclosporine A (CsA) had a higher response rate (83.7%) for severe aplastic anemia (SAA) than non-intensive immunosuppressive therapy (NIIST) with ALG alone (57.6%). IIST also resulted in a lower risk of early mortality and a shorter time to red cell transfusion independence.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : Chinese

6.

[Sequential intensified immunosuppressive therapy combining with hematopoietic growth factors in the treatment of severe aplastic anemia].

Author: HeG, ShaoZ, ZhangY, LiuH, LiK, SongL, ZhengY, ChenG, HeH, ZhaoM, ShiJ, ZhangH, ChuY, QianL, YangT, YangC

Severe aplastic anemia treatment

The addition of hematopoietic growth factors (HGFs) to sequential intensified immunosuppressive therapy (SIIST) for severe aplastic anemia (SAA) significantly improved response rates and reduced early infection and mortality rates.

English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : Chinese

7.

Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia.

Author: FrickhofenNorbert, HeimpelHermann, KaltwasserJoachim P, SchrezenmeierHubert,

Cyclosporine effective anemia
Salvage treatment effective
Remission unstable
Secondary disease common

Cyclosporine treatment in patients with aplastic anemia led to a significantly higher response rate (70% vs 41%) compared to treatment without cyclosporine. Patients with cyclosporine also had a faster response time (median 60 vs 82 days) and required fewer courses of immunosuppression. While overall survival was similar between groups, failure-free survival favored the cyclosporine regimen (39% vs 24%). However, relapse rates were similar and remissions were cyclosporine dependent in 26% of patients.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

8.

Immunosuppressive therapy for acquired severe aplastic anemia (SAA): a prospective comparison of four different regimens.

Author: ZhengYizhou, LiuYongze, ChuYulin

Hematopoietic recovery
Increased survival
Early deaths

The combination of equine anti-human thymocyte immunoglobulin (E-ATG) and cyclosporine A is the most effective treatment for acquired severe aplastic anemia, resulting in a survival advantage at 5 years.

Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

9.

[Efficacy of immunosuppressive therapy in children with acquired aplastic anemia].

Author: WangShu-chun, ZouYao, ChenXiao-juan, YangWen-yu, LiuTian-feng, ZhangLi, ChenYu-mei, GuoYe, ZhuXiao-fan

Improved SAA survival
None reported

This study found that the combination of R-ATG and CSA remains the best treatment for children with SAA who have no HLA-matched sibling, providing a survival advantage at 5 years.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : Chinese

10.

First-line matched related donor hematopoietic stem cell transplantation compared to immunosuppressive therapy in acquired severe aplastic anemia.

Author: PeinemannFrank, GrouvenUlrich, KrögerNicolaus, BartelCarmen, PittlerMax H, LangeStefan

SAA treatment

Acquired severe aplastic anemia (SAA) is a rare disease where the immune system attacks blood-forming stem cells. If a matched donor is available, a stem cell transplant is the preferred treatment. Immunosuppressive therapy is an alternative. This study compared the outcomes of these two treatments in controlled trials.

Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.

Language : English

11.

First-line allogeneic hematopoietic stem cell transplantation of HLA-matched sibling donors compared with first-line ciclosporin and/or antithymocyte or antilymphocyte globulin for acquired severe aplastic anemia.

Author: PeinemannFrank, BartelCarmen, GrouvenUlrich

Aplastic anemia treatment

Acquired severe aplastic anemia is a rare and potentially fatal blood disorder characterized by a lack of blood cells due to a problem with the bone marrow. Symptoms include infections, bleeding, and fatigue. The primary treatment is a stem cell transplant from a matching sibling donor. If a matching sibling is not available, medications like ciclosporin or antithymocyte/antilymphocyte globulin are used to suppress the immune system.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

12.

A systematic review and meta-analysis of the safety and efficacy of anti-thymocyte globulin combined with eltrombopag in the treatment of severe aplastic anemia.

Author: ZhangJiayou, WuYabing, LiuJunhui, HanShaolin, ChenLijuan, WangHui, PengYu

Aplastic anemia treatment

Aplastic anemia is a serious blood disorder. The standard treatment often combines anti-thymocyte globulin (ATG) and eltrombopag. While this combination has shown positive results, we need more research to confirm its long-term safety.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

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