# Treatment options for aplastic anaemia
The treatment of aplastic anaemia is tailored to the severity of the disease, the patient's age and the available donor material. The main strategies focus on restoring bone marrow function and combating the immune-mediated process that underlies many cases. Below is an overview of standard treatments by phase.
First line: supportive care
Supportive care forms the basis for all forms of aplastic anaemia. This includes regular blood transfusions (red blood cells and platelets), treatment of infections and nutrition, preferably in close collaboration with haematological centres. These measures prevent acute complications, but do not resolve the underlying problem and are therefore combined with active treatments.
Immunosuppressive therapy (IST)
ProveniIncluded in official guidelines, or approved by EMA or FDA
In many patients with acquired aplastic anaemia, the immune system plays a role: the body's own immune cells damage bone marrow cells. Immunosuppressive therapy targets this harmful immune reaction. Standard treatment consists of horse antithymocyte globulin (ATG), often combined with cyclosporine. These medicines suppress T-cells and other immune processes that attack the bone marrow.
ATG works by binding to certain immune cells, causing them to be broken down or become inactive. Cyclosporine prevents certain immune cells from becoming active. Together they provide some form of recovery in approximately 70% of patients; however, complete recovery is rare.
Side effects of ATG include fever, headache, muscle pain and noisy reactions during infusion. Cyclosporine can affect the kidneys, liver and nervous system. Risk of infection increases. Regular monitoring and checks are necessary.
Immunosuppressive therapy is usually given in the early stage of the disease, especially in patients without suitable donor material or in whom stem cell transplantation is not medically appropriate.
Thrombopoietin receptor agonists
ProveniIncluded in official guidelines, or approved by EMA or FDA
Medicines that activate the thrombopoietin receptor (TPO-RAs) stimulate the bone marrow to produce more platelets and can also affect red blood cell formation. The most studied substances in this class are eltrombopag and romiplostim. These agents can be used as first-line treatment, usually in combination with immunosuppressive therapy, or when IST is not sufficiently effective.
Eltrombopag is a small molecule that can be taken orally; romiplostim must be injected. Both inhibit immune attack on the bone marrow and stimulate stem cell growth.
Much research data from 2026 points to long-term beneficial effects when TPO-RAs are added early on, particularly in achieving normalisation of blood counts and sustained transfusion independence. Side effects are generally mild: headache, fatigue, abdominal pain. With long-term use, caution is warranted because of a theoretical risk of anaemia due to bone marrow fibrosis, although this is rare in practice.
Stem cell transplantation (allogeneic)
ProveniIncluded in official guidelines, or approved by EMA or FDA
For patients with suitable donor material — ideally an identical twin or HLA-matched blood relative or unrelated donor — allogeneic stem cell transplantation (alloSCT) offers the best chance of complete cure. In this procedure, the patient's damaged bone marrow is replaced by healthy blood-forming stem cells from a donor.
AlloSCT usually begins with preparation (conditioning) using chemotherapy and/or radiation, to eliminate the remaining diseased bone marrow and suppress the patient's immune system so that donor cells can engraft. The donor cells are then infused. The donor's immune cells develop the patient's diseased bone marrow and usually engraft well.
The main risks are:
- *Graft rejection*: the patient rejects the donor cells.
- *Graft-versus-host disease (GVHD)*: the donor cells recognize the patient's body as 'foreign' and attack it, particularly the skin, liver, and gastrointestinal tract.
- *Infections* due to reduced immunity during the engraftment period.
- *Secondary malignancies* in the long term.
When the engraftment succeeds, blood counts usually recover completely. This is why alloSCT is the preferred choice for many younger patients and fit enough older patients (typically up to around 65 years, depending on performance status).
Recent studies (2026) investigate improvements in pre-transplant stages (pre-transplant support with TPO-RAs) and reduction of GVHD complications, including through tissue transplantation in severe liver damage after transplantation.
Intensive chemotherapy (alternative)
ProveniIncluded in official guidelines, or approved by EMA or FDA
In patients who do not have a suitable donor and cannot undergo stem cell transplantation, intensive chemotherapy (for example with cyclophosphamide, possibly in combination with fludarabine) can sometimes stimulate the bone marrow. This approach is used much less frequently than IST, because results are more cautious and the treatment can be more demanding.
Drugs that target specific cellular attacks
ResearchediPositive results in clinical studies, not yet standard treatment
New research (2026) focuses on more targeted approaches. For example, some agents work on specific signalling pathways involved in immune attack on bone marrow cells — such as the STING pathway or metabolic adaptations. These are not yet routine, but are being tested in clinical trials as a complement to or replacement for classical IST.
Cure without chemotherapy (targeted recovery)
ResearchediPositive results in clinical studies, not yet standard treatment
Experimental techniques investigate the use of guided stem cell or stem cell derivatives prepared in the laboratory (for example, in vitro expanded stem cells) to restore the bone marrow 'gently', without full chemotherapy burden. In the future, this may be particularly interesting for elderly patients and those with medical conditions. Umbilical cord transplantation with pre-mobilized cells is an example of targeted research.
Maintenance and follow-up treatment
After achieving response (with IST, TPO-RAs or transplantation), many patients receive long-term monitoring and sometimes maintenance medication to prevent relapse. Immunosuppressive medication can be cautiously tapered. Infection prophylaxis and regular blood tests may continue to be necessary.
When patients relapse (the bone marrow fails again) after IST, they can undergo IST again, be treated with TPO-RAs, or may still be candidates for stem cell transplantation — a decision tailored to age, condition and available options.
Hereditary forms
In patients with hereditary bone marrow diseases (such as Fanconi anaemia), the treatment approach is more cautious. Chemotherapy may carry additional risks. Stem cell transplantation may be beneficial, but preparation sometimes needs to be lighter. Regular screening for additional disorders and cancer screening is important.
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_This information never replaces a doctor's judgment. Always discuss your situation with your own healthcare provider._