Exploring A Patient’s Journey with Malignant PEComa

Cristina is an actual patient. She is a paid consultant of Aadi Bioscience, Inc.

Introduction to Cristina

As a passionate dancer, Cristina enjoyed an active life of dancing jazz, musical theater, Latin and belly dancing for many years. However, after a routine visit to her doctor in 2013, Cristina’s world began to change dramatically. Over several years, Cristina’s journey took her through multiple diagnoses, numerous surgeries, and rounds of chemotherapy with challenging side effects. However, Cristina was resilient, always focusing on the positive aspects of her life.

Cristina’s Initial Diagnosis

At an annual visit to her gynecologist, Cristina described a painless, swelling-like discomfort in her abdomen. Cristina's gynecologist decided to perform a sonogram of her uterus to investigate potential causes of her symptoms. Results from the sonogram revealed a large tumor mass in Cristina’s uterus.

For tumor removal, a laparoscopic hysterectomy was performed, in which the uterus was surgically removed using several small incisions in the lower abdomen.¹ A tumor sample was sent out for further laboratory testing after the surgery, which came back to show that this was a rare tumor type that could potentially be cancerous. Remembering the shock and emotion she felt, Cristina recalled, “I was thinking, well, how can that be? I’m healthy, I dance.”

For a more thorough analysis, Cristina’s gynecologist also sent a sample to a specialty laboratory. After about a week of anxiously awaiting the results, Cristina’s gynecologist called and shared that the tumor was benign with no apparent cancer risk. “Then with feelings of relief I’m thinking, oh, yay, hooray,” Cristina recalled.

Symptoms Return

After two worry-free years thinking that her tumor was benign, Cristina’s abdominal symptoms reemerged, and she returned to her gynecologist for a follow-up sonogram. Much to her dismay, the sonogram revealed multiple tumors. At this time she was diagnosed with endometrial sarcoma and she learned that her initial diagnosis was not benign, but a low-grade sarcoma. As a result, Cristina was referred to a surgical oncologist to undergo cytoreduction, also referred to as debulking surgery, an operation to remove as much cancer as possible from the body.²

Following debulking surgery, Cristina was referred to an oncologist who then initiated a carboplatin-based chemotherapy regimen to eliminate any residual tumor tissue. Initially, Cristina received chemotherapy via an inner abdominal port and later via a peripheral intravenous line in her arm. However, repeated chemotherapy doses eventually caused enough damage to the blood vessels in her arms that a central chest port was placed.³ Cristina also experienced significant numbness (neuropathy) in her feet, which continues to plague her to this day, making it challenging to maintain balance.

Relapse Following Chemotherapy and New Diagnosis

After two years of chemotherapy treatment, follow-up CT scans showed that all the tumors had significantly decreased in size, and it was beginning to look like Cristina may be on the road to remission. Consequently, Cristina’s oncologist decided to pause chemotherapy for a watch and wait period. However, Cristina’s hope was shattered after a few months, a follow-up CT scan revealed the enlargement of existing tumors and the emergence of new lesions.

With treatment options exhausted, Cristina was referred to a surgeon who specialized in gynecological oncology. Upon initial inspection of the tumor sample, Cristina’s surgeon felt the sample was consistent with a rare type of cancer, known as malignant perivascular epithelioid cell tumor (PEComa). For diagnosis confirmation and further evaluation of the tumor, a tissue sample was sent for additional testing. Results confirmed the presence of malignant PEComa and revealed genetic information about the tumor.

Malignant PEComa is a certain type of cancer known as a soft tissue sarcoma, which is a rare type of cancer that forms in soft tissues, including fat, blood vessels, muscles, and nerves.⁴

Treatment Options for Malignant PEComa

Malignant PEComa is a type of cancer that can spread throughout the body. If a malignant PEComa is found in the early stages, it may be surgically removed with no further treatment.⁵ Locally advanced or metastatic malignant PEComa refers to malignant PEComa that cannot be removed by surgery and/or has spread (metastasized) to other parts of the body.⁶ While there is no cure for advanced malignant PEComa, it is treatable.^5,7

Due to the rare incidence of malignant PEComa and the advanced stages of Cristina’s cancer, treatment options were limited. Fortunately, a clinical trial investigating the use of a drug called nanoparticle albumin-bound (nab)-sirolimus—also known as FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound)—in patients with advanced malignant PEComa was ongoing at the time of Cristina’s diagnosis. Based on tumor findings, Cristina’s oncologist decided that FYARRO would be the best treatment option.⁸ FYARRO is a prescription medicine used to treat locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumors in adult patients.⁹

Please see full Prescribing Information and Important Safety Information at the end of this article for FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound).

Navigating Steps With Cristina’s Healthcare Team

Before starting treatment, Cristina’s oncologist explained that FYARRO is different from chemotherapy drugs because, based on studies in animals, it can target cancer cells to a greater extent than other cells in the body.⁹

Cristina received treatment with FYARRO for approximately four years. Recalling her experience while receiving FYARRO, Cristina expressed, “it only took half an hour to [infuse]” and the nurses at the infusion center were “absolutely fabulous.” FYARRO is given as an intravenous infusion, which means that the medication is given directly and slowly into the blood through a vein, usually in the arm. Each infusion of FYARRO takes 30 minutes. FYARRO is given two times during a 3-week period.⁹

Cristina’s tumor responded well to treatment with FYARRO with a significant size reduction seen in all of her tumors. After a few years of treatment, there was Cristina’s tumors had nearly disappeared, and her hope was once more renewed. Cristina was finally able to receive a diagnosis that helped guide her care team to a treatment designed to battle her specific type of cancer.

Managing Side Effects

Although Cristina experienced some side effects during treatment (eg, nausea, rash, decrease in appetite, and weight loss), they were mild compared to those experienced with chemotherapy. Additionally, Cristina's oncologist prescribed her oral medications to manage episodes of nausea and topical creams to manage her rash.

Before beginning treatment with FYARRO, it is important to know about the possible side effects and precautions, and address them with your healthcare provider if they occur. The most common side effects with FYARRO include: mouth ulcers or sores, feeling weak or tired, rash, infection, nausea, swelling of arms, hands, feet, ankles, face, or other parts of the body, diarrhea, body pain, decreased weight, decreased appetite, cough, vomiting, and altered taste.⁹ Serious side effects occurred in 14 patients (41%) who received FYARRO. Most common serious side effects were infection, stomach pain, dehydration, and upper gastrointestinal hemorrhage.⁹

Click here for a list of potential side effects that may be experienced with FYARRO treatment.

Cristina’s Advice for Others

Many emotions can accompany a cancer diagnosis, and it can be helpful to surround yourself with positive and supportive people. “It’s OK to cry and go through all those emotions,” Cristina reassured. “Find a close friend that you can talk to and confide in,” she continued. “They can sometimes be more objective than somebody close to you.”

Cristina encouraged other patients to ask their healthcare team about genetic testing for mutations that may be associated with malignant PEComas.⁴ Cristina emphasized the importance of reviewing their diagnostic reports with their provider to understand the full scope of their diagnosis and treatment plan. “Some people are very tentative around doctors—[they call it a ‘white coat syndrome’]. Don't be afraid. You are your own advocate. Look at all the [diagnostic reports, scans, and] read everything.”

Cristina further shared how helpful it can be to find activities that take your mind off your diagnosis, stating, “Listen to music, find a hobby, find something that you are passionate about.” Sometimes, this means focusing on what you can do. “I couldn’t dance, so what else can I do?” Cristina recalled, “Well, I sew, and I love to paint, there’s always something. Find something that you love, that you can do. Don’t concentrate on what you can’t do.”

While this is Cristina's own experience with malignant PEComa and treatment with FYARRO, it is important to understand that each patient and their journey with this disease is different and patients should work closely with their health care teams to determine an appropriate plan for managing their malignant PEComa.

Additional resources are available here.

Indication

FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) is a prescription medicine used to treat locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumors in adult patients.

Important Safety Information

How is FYARRO taken?
FYARRO is administered by intravenous infusion into your vein over 30 minutes on Days 1 and 8 of each 21-day cycle until disease progression or unacceptable toxicity.

Who should not receive FYARRO?

Do not use FYARRO if you have had a severe allergic reaction to sirolimus or human albumin. You should tell your health care provider before receiving FYARRO if you are allergic to sirolimus (Rapamune®), everolimus (Afinitor®), temsirolimus (Torisel®) or Human Albumin.

What are the possible side effects of FYARRO?

FYARRO can cause serious side effects, including the following:

Mouth ulcers and sores

Mouth ulcers and sores are common during treatment with FYARRO and can be severe. When you start treatment with FYARRO, your health care provider may also prescribe an alcohol-free mouthwash to reduce the likelihood of getting mouth ulcers or sores and to reduce their severity. Follow your health care provider’s instructions on how to use this prescription mouthwash. If you develop pain, discomfort, or open sores in your mouth, tell your health care provider. Your health care provider may tell you to restart this mouthwash or to use a special mouthwash or mouth gel that does not contain alcohol, peroxide, iodine, or thyme.

Decreased blood cell counts

FYARRO can cause you to have decreased red blood cells, platelets, and white blood cells. Your health care provider should do blood tests to check your blood cell counts before you start and during treatment with FYARRO.

Infections

Infections such as pneumonia, or a bacterial, fungal, or viral infection may develop while on treatment with FYARRO. Viral infections may include active hepatitis B in people who have had hepatitis B in the past (reactivation). In some people (including adults and children), these infections may be severe and can lead to death. If infections occur, urgent treatment may be required. Tell your health care provider right away if you have a temperature of 100.5°F or above, have chills, or do not feel well. Symptoms of hepatitis B or infection may include the following: fever, chills, skin rash, joint pain and swelling, tiredness, loss of appetite, nausea, pale stools or dark urine, yellowing of the skin, or pain in the upper right side of the stomach.

Low potassium

FYARRO can cause your blood potassium levels to fall. Low blood potassium levels can make you feel weak and can affect your heart rhythm if severe. Your health care provider should do blood tests to check your potassium levels before you start and during treatment with FYARRO.

Increased blood sugar and fat (cholesterol and triglyceride) levels in blood

Your health care provider should do blood tests to check your fasting blood sugar, cholesterol, and triglyceride levels in the blood before you start and during treatment with FYARRO. Diabetic patients should monitor their fasting serum glucose more frequently.

Lung inflammation

FYARRO can cause inflammation of the lung. If you develop a persistent cough, chest pain, or shortness of breath, let your doctor know right away.

Bleeding

FYARRO can cause serious and sometimes fatal bleeding. If you develop bleeding, let your doctor know right away.

Severe allergic reactions

Call your health care provider or get medical help right away if you get signs and symptoms of a severe allergic reaction including rash, itching, hives, difficulty breathing or swallowing, flushing, chest pain, or dizziness.

Harm to an unborn child

FYARRO can cause harm to your unborn baby. For women who are able to become pregnant, your health care provider will give you a pregnancy test before you start treatment with FYARRO. You should use effective birth control during treatment and for 12 weeks after your last dose of FYARRO. Men with a female partner should use effective birth control during treatment and for 12 weeks after your last dose of FYARRO.

Infertility

FYARRO may affect fertility in woman and men and may affect your ability to become pregnant if you are female, or your ability to father a child if you are male. Talk to your health care provider if this is a concern for you.

Breastfeeding

Do not breastfeed during treatment and for 2 weeks after your last dose.

Vaccinations

You should not receive a live vaccine or be around people who have recently received a live vaccine during your treatment with FYARRO. If you are not sure about the type of vaccine, ask your health care provider.

Human albumin

Human serum albumin (HSA), an ingredient in FYARRO, is made from human blood. The blood donors for HSA are checked for infections and the HSA is processed to get rid of germs in the product. Because of these steps, HSA carries only an extremely small risk for transmission of viral diseases and “mad cow” disease, also called variant Creutzfeldt-Jakob disease (vCJD). No cases of transmission of viral diseases, CJD, or vCJD have ever been identified for licensed albumin.

The most common side effects with FYARRO include the following:

• Mouth ulcers or sores
• Feeling weak or tired
• Rash
• Infection
• Nausea
• Swelling of arms, hands, feet, ankles, face, or other parts of the body
• Diarrhea
• Body pain
• Decreased weight
• Decreased appetite
• Cough
• Vomiting
• Altered taste

Other possible side effects that may occur with FYARRO:

• Absence of menstrual periods (menstruation). You may miss 1 or more menstrual period. Tell your health care provider if this happens

What should I tell my health care provider before taking FYARRO?

Before taking FYARRO, tell your health care provider about all your medical conditions, including if you:

• Have or have had kidney problems
• Have or have had liver problems
• Have diabetes or high blood sugar
• Have high blood cholesterol levels
• Have any infections
• Previously had hepatitis B

Tell your health care provider about all of the medicines you take, including prescription and nonprescription medicines, vitamins, and herbal supplements. Using FYARRO with certain other medicines can cause serious side effects. Keep a list of medicines you take and show it to your health care provider when you get a new medicine. Especially tell your health care provider if you take the following:

• St John’s wort (Hypericum perforatum)
• Medicine for:
— Fungal infections
— Bacterial infections
— Tuberculosis
— Seizures
— HIV-AIDS
— Heart conditions or high blood pressure
• Medicines that weaken the immune system (your body’s ability to fight infections and other diseases)

Ask your health care provider or pharmacist if you are not sure if your medicine is one of those taken for the conditions listed above. If you are taking any medicines for the conditions listed above, your health care provider might need to prescribe a different medicine or your dose of FYARRO may need to be changed. Tell your health care provider before you start taking any new medicine.

What should I do if I receive FYARRO from my health care provider?

You should not drink grapefruit juice or eat grapefruit during your treatment with FYARRO. It may make the amount of FYARRO in your blood increase to a harmful level.

Tell your health care provider if you have any side effect that bothers you or does not go away.

These are not all the possible side effects of FYARRO. For more information, ask your health care provider or pharmacist. Call your doctor for medical advice about side effects.

The risk information provided here is not comprehensive. To learn more, talk about FYARRO with your health care provider or pharmacist. The FDA-approved product labeling can be found at www.FYARRO.com or 1-888-BIO-AADI.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Important Safety Information above in this article and full Prescribing Information.

References

1. Laparoscopic hysterectomy. The Permanente Medical Group; 2005. Accessed June 30, 2022. https://thrive.kaiserpermanente.org/care-near-you/northern-california/eastbay/wp-content/uploads/sites/6/2015/10/Laparoscopic-Hysterectomy_tcm28-461219.pdf
2. Debulking surgery/cytoreduction. Cancer Treatment Centers of America; 2022. Accessed June 30, 2022. https://www.cancercenter.com/treatment-options/surgery/debulking-surgery
3. What is a chemo port? Moffitt Cancer Center. Accessed June 30, 2022. https://moffitt.org/treatments/chemotherapy/what-is-a-chemo-port/
4. About soft tissue sarcomas. American Cancer Society. Accessed July 29, 2022. https://www.cancer.org/content/dam/CRC/PDF/Public/8813.00.pdf
5. Tirumani SH, Shinagare AB, Hargreaves J, et al. Imaging features of primary and metastatic malignant perivascular epithelioid cell tumors. AJR Am J Roentgenol. 2014;202(2):252-258. doi:10.2214/AJR.13.10909
6. Treating soft tissue sarcomas. American Cancer Society. Accessed July 29, 2022. https://www.cancer.org/content/dam/CRC/PDF/Public/8816.00.pdf
7. Ben-Ami E, Hornick JL, Wagner AJ. The potential of emerging new therapeutics for the treatment of perivascular epithelioid cell tumors (PEComa). Expert Opin Orphan Drugs. 2018;6(9):537-543. doi:10.1080/21678707.2018.1493376
8. A phase 2 study of ABI-009 in patient with advanced malignant PEComa (AMPECT). ClinicalTrials.gov. Updated May 3, 2022. Accessed June 30, 2022. https://clinicaltrials.gov/ct2/show/NCT02494570
9. FYARRO [sirolimus protein-bound particles for injectable suspension (albumin-bound)]. Prescribing Information. Aadi Biosciences, Inc.; 2021. Accessed June 30, 2022. https://fyarro.com/images/prescribing/prescribing-information.pdf

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