The Critical Role of Anti-Rejection Medication
Following an organ transplant, the recipient's immune system recognizes the new organ as a foreign invader and mounts an attack, a process known as rejection [1.11.4]. To prevent this, patients must take a lifelong regimen of drugs called immunosuppressants, or anti-rejection medications [1.11.3]. These powerful drugs work by weakening the immune system just enough to allow the body to accept the donor organ without leaving the patient completely vulnerable to infections [1.4.2]. The development of these medications has dramatically improved transplant outcomes, with acute rejection rates for kidney transplants, for example, dropping from over 50% in the 1970s to around 10-20% today [1.2.3].
Immunosuppressive therapy is typically divided into three phases [1.11.1]:
- Induction Therapy: This involves a potent course of medication administered right before, during, or immediately after the transplant surgery [1.8.2]. The goal is to provide a strong level of immunosuppression when the risk of acute rejection is highest [1.7.2]. Agents like basiliximab or anti-thymocyte globulin are often used [1.3.2].
- Maintenance Therapy: This is the lifelong, daily regimen of drugs that transplant recipients must take to prevent rejection over the long term [1.7.1]. It usually involves a combination of several drugs from different classes to maximize effectiveness and minimize side effects [1.7.2].
- Treatment of Rejection: If the body begins to reject the organ despite maintenance therapy, higher doses or different types of immunosuppressants are used to stop the rejection episode [1.11.1].
Major Classes of Immunosuppressant Drugs
Maintenance therapy typically combines drugs from at least two or three of the following classes to target the immune system in different ways [1.4.5, 1.7.2].
Calcineurin Inhibitors (CNIs)
This class is a cornerstone of most anti-rejection regimens [1.4.5]. CNIs work by blocking a protein called calcineurin, which in turn inhibits the production of interleukin-2, a substance that activates T-cells—the primary drivers of organ rejection [1.4.4].
- Examples: Tacrolimus (Prograf®, Astagraf XL®, Envarsus XR®) and Cyclosporine (Neoral®, Sandimmune®, Gengraf®) [1.3.5].
- Side Effects: CNIs can be tough on the kidneys (nephrotoxicity), and may also cause high blood pressure, high cholesterol, tremors, and an increased risk of developing diabetes after transplant [1.5.2, 1.6.3]. Tacrolimus is generally considered more potent but may have a higher incidence of new-onset diabetes compared to cyclosporine [1.6.3].
Antiproliferative Agents (Antimetabolites)
These drugs work by preventing immune cells like T-cells and B-cells from multiplying [1.11.1]. They are almost always used in combination with a CNI [1.4.5].
- Examples: Mycophenolate Mofetil (CellCept®), Mycophenolic Acid (Myfortic®), and Azathioprine (Imuran®) [1.3.5].
- Side Effects: The most common side effects are gastrointestinal, including diarrhea, nausea, and stomach upset [1.5.2]. They can also lower white blood cell and platelet counts [1.5.2]. Mycophenolate products are known to increase the risk of birth defects and pregnancy loss [1.3.5].
mTOR Inhibitors
Named for their mechanism of inhibiting the 'mammalian target of rapamycin,' these drugs block a different pathway involved in T-cell activation and proliferation [1.6.1]. They are often used as an alternative for patients who cannot tolerate the kidney-related side effects of CNIs [1.3.5].
- Examples: Sirolimus (Rapamune®) and Everolimus (Zortress®) [1.3.5].
- Side Effects: Common side effects include high cholesterol and triglycerides, delayed wound healing, mouth sores, and swelling [1.5.2].
Corticosteroids
These are powerful anti-inflammatory drugs that suppress the immune system in a broad, non-specific way [1.4.4].
- Example: Prednisone [1.3.1].
- Side Effects: While effective, long-term use is associated with many side effects, including weight gain, mood swings, high blood sugar, high blood pressure, osteoporosis (bone loss), and cataracts [1.5.2, 1.10.4]. Due to these effects, many transplant centers aim to reduce or eliminate steroid use over time.
Comparison of Common Maintenance Drugs
Drug Class | Common Drugs | Mechanism of Action | Key Side Effects |
---|---|---|---|
Calcineurin Inhibitors | Tacrolimus, Cyclosporine | Inhibit T-cell activation by blocking calcineurin [1.4.4] | Kidney damage, high blood pressure, diabetes, tremors [1.6.3] |
Antiproliferatives | Mycophenolate, Azathioprine | Prevent multiplication of immune cells [1.11.1] | Diarrhea, nausea, low white blood cell count [1.5.2] |
mTOR Inhibitors | Sirolimus, Everolimus | Block a key protein (mTOR) for cell proliferation [1.6.1] | High cholesterol, mouth sores, poor wound healing [1.5.2] |
Corticosteroids | Prednisone | Broad anti-inflammatory and immune suppression [1.4.4] | Weight gain, high blood sugar, bone loss, mood swings [1.5.2] |
Living with Immunosuppressants
Life after transplant requires strict adherence to the prescribed medication schedule. Missing even a single dose can increase the risk of rejection [1.11.3]. Patients must also be vigilant for side effects and signs of infection, as a suppressed immune system has a harder time fighting off common illnesses [1.5.4].
The financial burden can also be significant. Without insurance, these life-saving medications can cost between $5,000 and $7,000 per month [1.9.1, 1.9.2]. Even with coverage, co-pays can be substantial.
Regular blood tests are essential to monitor drug levels in the body. The goal is to find a balance: enough medication to prevent rejection but not so much that it causes severe side effects or dangerously high levels of immunosuppression [1.5.4].
Conclusion
To answer the question, "What drug do you take to prevent rejection of a donor organ?", there isn't just one. Patients take a carefully balanced, lifelong combination of immunosuppressant drugs. The mainstays of modern therapy include calcineurin inhibitors like tacrolimus, antiproliferative agents like mycophenolate, and often corticosteroids like prednisone [1.3.1, 1.3.2]. These medications are the key to turning organ transplantation from an experimental procedure into a life-saving treatment, allowing hundreds of thousands of people to live longer, healthier lives.
For more information from an authoritative source, you can visit the National Kidney Foundation's guide on Immunosuppressants. [1.3.5]