Pheochromocytoma is a rare tumor that develops in the center of the adrenal gland, producing excessive amounts of stress hormones that can cause dangerous spikes in blood pressure, severe headaches, and sweating. While this condition can lead to serious complications if left untreated, early detection and proper treatment can successfully control symptoms and, in many cases, cure the disease entirely.

How Doctors Approach Treating Pheochromocytoma

The main goal when treating pheochromocytoma is to remove the tumor and restore normal blood pressure, which helps patients return to their everyday lives. Because these tumors produce excessive amounts of hormones called catecholamines—including adrenaline and noradrenaline—treatment must carefully address both the tumor itself and the effects of these powerful hormones on the body. The treatment plan depends on several factors, including the size and location of the tumor, whether it has spread to other parts of the body, and the patient’s overall health.^[1]

Most pheochromocytomas are benign, meaning they are not cancerous, and usually affect only one of the two adrenal glands. However, approximately 10% to 15% of these tumors can be malignant, or cancerous, and may spread to other organs such as the liver, lungs, or bones. This makes accurate diagnosis and timely treatment essential. Once a pheochromocytoma is diagnosed, it is strongly recommended that patients work with a multidisciplinary medical team that has experience managing this rare condition.^[2][6]

Standard medical guidelines recommend that all pheochromocytomas be removed surgically whenever possible, as this is the only way to cure the disease. Before surgery can safely take place, however, doctors must first stabilize the patient’s blood pressure and prepare the body to handle the stress of the operation. This preparation phase is just as important as the surgery itself, because the tumor’s hormones can cause dangerous blood pressure swings during the procedure.^[3]

Standard Treatment for Pheochromocytoma

The cornerstone of treating pheochromocytoma is surgical removal of the tumor, a procedure known as adrenalectomy. This operation typically removes the entire affected adrenal gland along with the tumor. In most cases, surgery successfully returns blood pressure to normal and eliminates the symptoms patients have been experiencing. However, surgery cannot be performed immediately after diagnosis—careful preparation is needed first.^[1][8]

Preparing for Surgery with Medication

Before surgery, patients must take medications for approximately 10 to 14 days to control their blood pressure and protect their heart. This preparation phase is critical because the tumor releases large amounts of hormones that can cause life-threatening blood pressure spikes during surgery. The most commonly used medication is phenoxybenzamine, which belongs to a class of drugs called alpha blockers. These medications work by blocking the effects of adrenaline and noradrenaline on blood vessels, allowing them to relax and reducing blood pressure.^[15]

Phenoxybenzamine is typically started at a low dose and gradually increased over several days. The goal is to achieve stable blood pressure control while also allowing the body’s blood volume to expand naturally. Patients are often encouraged to increase their salt intake during this time, which helps the body retain more fluid and prevents blood pressure from dropping too low after surgery. Some patients may also need to receive intravenous fluids before the operation to ensure adequate blood volume.^[3][15]

After alpha blockade has been established for at least two days, doctors may add a beta blocker medication to control the heart rate. This timing is extremely important—if beta blockers are started too early, before adequate alpha blockade, they can cause a dangerous rise in blood pressure due to unopposed stimulation of blood vessels. Beta blockers help prevent rapid or irregular heartbeats during and after surgery. Patients take their last doses of both alpha and beta blockers on the morning of their operation.^[15]

The Surgical Procedure

Most pheochromocytomas are removed using laparoscopic adrenalectomy, a minimally invasive surgical technique. During this procedure, surgeons make several small incisions and use special instruments and a camera to remove the tumor and affected adrenal gland. This approach offers several advantages over traditional open surgery, including less pain, smaller scars, shorter hospital stays, and faster recovery. Some medical centers also offer a specialized technique called mini-back scope surgery, which approaches the adrenal gland from the patient’s back, potentially offering even faster recovery.^[7][15]

In certain situations, open surgery may be necessary instead of laparoscopic surgery. This is more likely when the tumor is very large, when there are multiple tumors, or when the cancer has spread beyond the adrenal gland. During surgery, the anesthesia team closely monitors blood pressure and heart function, as manipulating the tumor can cause sudden hormone releases. Having experienced surgeons and anesthesiologists who understand pheochromocytoma is crucial for a safe outcome.^[15][16]

Recovery and Follow-Up Care

After successful surgery, most patients experience immediate improvement in their symptoms, though it may take several weeks for blood pressure to normalize completely. Doctors typically test blood levels of metanephrines—substances created when the body breaks down catecholamines—about two weeks after surgery to confirm that the tumor was completely removed. If these levels return to normal, it indicates successful treatment. However, long-term follow-up is essential because pheochromocytomas can recur, meaning they can grow back or appear in the other adrenal gland. Some patients may need to have blood or urine tests annually for several years after surgery.^[11][13]

If a patient had only one adrenal gland removed, the remaining gland usually produces enough hormones for normal body function. However, patients who have both adrenal glands removed will need to take hormone replacement medications for the rest of their lives to replace the hormones their bodies can no longer make naturally.^[12]

Treatment for Malignant Pheochromocytoma

When a pheochromocytoma is malignant and has spread to other parts of the body, treatment becomes more complex. Surgery may still be performed to remove as much tumor tissue as possible, which can help reduce symptoms even if it cannot cure the cancer. Other treatment options for malignant pheochromocytoma include radiation therapy, which uses high-energy rays to destroy cancer cells, and chemotherapy, which uses drugs to kill rapidly dividing cancer cells throughout the body.^[8][13]

Some patients with metastatic pheochromocytoma may also receive treatment with radioactive compounds that are specifically taken up by the tumor cells. This type of treatment delivers radiation directly to the cancer while sparing healthy tissue. Additionally, medications may be used long-term to control blood pressure and other symptoms caused by excess hormone production.^[11]

Treatment in Clinical Trials

For patients whose pheochromocytomas cannot be completely removed with surgery or for those with malignant tumors that have spread, participating in clinical trials offers access to promising new treatments that are still being studied. These research studies test innovative approaches that may become standard treatment options in the future.

Targeted Radiation Therapy

One area of active research involves specialized forms of radiation treatment that target pheochromocytoma cells more precisely. A notable example is treatment with Lutetium-177 DOTATATE, a radioactive compound that binds to specific receptors on the surface of pheochromocytoma cells. This treatment has shown promise in clinical trials for progressive metastatic pheochromocytomas and paragangliomas. In a Phase II study, this therapy demonstrated the ability to slow tumor growth and control symptoms in patients whose disease had continued to progress despite other treatments. The radioactive compound delivers radiation directly to the tumor cells while causing less damage to surrounding healthy tissue than traditional external radiation therapy.^[5]

This type of treatment, called radiopharmaceutical therapy, represents an important advance because it can reach cancer cells wherever they are in the body, making it particularly useful for patients whose disease has spread to multiple locations. Patients receiving this treatment typically undergo several treatment cycles over several months, with careful monitoring between sessions to assess how well the treatment is working and to watch for side effects.^[15]

Novel Drug Therapies

Researchers are investigating several new medications that work by interfering with specific molecular pathways that pheochromocytoma cells need to survive and grow. One class of drugs being studied are hypoxia-inducible factor inhibitors. These medications target a protein that helps tumors adapt to low-oxygen conditions, which is important for their growth and spread. By blocking this protein, these drugs may slow or stop tumor growth. Clinical trials are testing the safety and effectiveness of these agents in patients with advanced pheochromocytoma.^[15]

Other investigational approaches include drugs that target the blood supply to tumors, known as anti-angiogenic therapies. These medications work by preventing tumors from forming new blood vessels, which they need to receive nutrients and oxygen. Without an adequate blood supply, tumors may shrink or stop growing. Several of these drugs are being evaluated in Phase II clinical trials, which assess whether they are effective in treating pheochromocytoma while also gathering more information about their safety.

Understanding Clinical Trial Phases

Clinical trials progress through different phases, each designed to answer specific questions. Phase I trials primarily focus on safety, testing a new treatment in a small group of people to determine safe dosing and identify side effects. Phase II trials involve more patients and evaluate whether the treatment actually works against the disease while continuing to monitor for side effects. Phase III trials compare the new treatment to standard treatments in large groups of patients to determine which approach is more effective. Understanding these phases helps patients know what to expect if they consider joining a trial.^[3]

Eligibility and Locations

Clinical trials for pheochromocytoma are being conducted at medical centers throughout the United States, Europe, and other regions. Patient eligibility depends on many factors, including the stage of disease, previous treatments received, overall health status, and specific characteristics of the tumor. Patients interested in clinical trials should discuss options with their medical team and can search for available studies through registries such as ClinicalTrials.gov. Participation in clinical trials not only provides access to cutting-edge treatments but also contributes to medical knowledge that will help future patients with this rare condition.^[5][6]

Most common treatment methods

• Surgical Treatment
  • Laparoscopic adrenalectomy, which uses small incisions and specialized instruments to remove the tumor and affected adrenal gland
  • Open surgical removal for larger tumors or when cancer has spread beyond the adrenal gland
  • Mini-back scope surgery, a specialized approach that accesses the adrenal gland from the patient’s back
• Preoperative Medical Management
  • Alpha blocker medications such as phenoxybenzamine to control blood pressure before surgery
  • Beta blocker medications to regulate heart rate and prevent palpitations
  • Intravenous fluid administration to expand blood volume and prevent low blood pressure after surgery
  • Other blood pressure medications including calcium channel blockers and angiotensin receptor blockers
• Treatment for Malignant Disease
  • Radiation therapy using high-energy rays to destroy cancer cells that have spread
  • Chemotherapy with drugs that target rapidly dividing cancer cells throughout the body
  • Radiopharmaceutical therapy with Lutetium-177 DOTATATE, which delivers targeted radiation to tumor cells
  • Long-term medication management to control blood pressure and symptoms from excess hormones
• Investigational Therapies in Clinical Trials
  • Hypoxia-inducible factor inhibitors that block proteins helping tumors adapt to low-oxygen conditions
  • Anti-angiogenic therapies that prevent tumors from forming new blood vessels
  • Novel targeted radiation treatments that deliver therapy more precisely to tumor cells