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Clinical Trials Awareness
Access to a clinical trial means more energy to bake cakes with her sister
Diagnosis: High risk pre b-cell acute ALL. Ph-
Age at Diagnosis: 4
Clinical Trial: Blinatumomab
Gabby was a fun and bouncy four-year-old who loved baking, reading, and spending time with her big sister. Just after her fourth birthday, Gabby became lethargic, pale, and had intense pain in her legs. She was having trouble breathing at night and her parents suspected pneumonia. However, Gabby’s breathing problems were because she was in heart failure, a symptom of leukemia. Gabby was diagnosed at the children’s hospital in London with acute lymphoblastic leukemia (ALL) on August 24, 2013. She was considered high-risk because her cancer had a white blood cell count of 259, far above the threshold of 10.
Because of the aggressiveness of Gabby’s cancer, her two-year frontline treatment involved an intensive chemotherapy schedule. Gabby encountered many complications. She had life-threatening allergic reactions and was in the ICU three times with infections. She didn’t eat and was in a lot of pain. There was hair loss, no physical strength, nausea, and vomiting.
In September 2015, after two years of chemotherapy treatment a routine lumbar puncture discovered leukemia cells isolated in her central nervous system. Gabby’s doctors suggested a clinical trial with the Children’s Oncology Group. The trial included two more years of chemotherapy, plus cranial radiation and introduced 3 rounds of the immunotherapy medication, blinatumomab. Blinatumomab is a monoclonal antibody that can selectively identify leukemia cells and direct the immune system to kill the cancerous cells.
The clinical trial was preparing to open in Canada, Gabby became patient one and was the first Canadian child to receive the treatment. The trial was randomized, but Gabby fell into the category of receiving blinatumomab. She received the same frontline treatment chemotherapy drugs as before, but in combination with blinatumomab.
After the first month of her treatment with chemotherapy, Gabby had a negative minimal residual disease (MRD), also known as no evidence of disease. After 6 months of chemotherapy, Gabby entered the next stage of her treatment, a 30 day, 24 hours a day, infusion of blinatumomab. She was admitted to hospital for 8 days, put into isolation and monitored for side-effects. She had a 24-hour nurse inside the room for the first 4 days. She experienced no negative effects, including cytokine release syndrome (CRS), where cancer cells release a toxin into the patient’s system which can cause side effects that can be as mild as a fever or as serious as multiple organ failure.
Because she was doing so well, Gabby’s team decided to switch her over to a portable pump to receive the blinatumomab infusion and allow her to be treated at home. Gabby’s family used a backpack to carry the pump, which would hold a 48-hour supply. Her parents were taught by the pediatric nurses how to ensure that the tubes were safe and to facilitate the 24-hour infusion at home.
Gabby’s mom videoed the process of ‘outfitting’ her with the pump, fanny pouch (with medicine and pump) and the backpack so that it could be used to help train oncology nurses at other trial sites across Canada. Gabby was outpatient on the 8
day. Her family would return with her to the hospital on a timed 48-hour cycle to ensure the bag was swapped out so that the blinatumomab infusion wasn’t interrupted.
The quality of life on blinatumomab was vastly superior to chemotherapy. Gabby was in a great mood, was active, energetic, and had an appetite. She was able to go to school and visit with friends as there were less concerns about infection. There were no mouth sores, no sleeping issues, no mood issues. She appeared to have the quality of life of a perfectly healthy child.
This experience was much different than her experience with chemotherapy. With chemotherapy, Gabby was largely isolated at home and couldn’t have time with extended family or friends, nor could she attend school regularly. Her family had to maintain a level of isolation to ensure that they were not bringing infections into the house. Her sister had limited social ability and her parents had to pull her from school if there were any infections going around. The isolation had a major impact on Gabby and her sister. They had frequent hospital stays due to infections, which would separate the family, and made it impossible for her mom to work.
With blinatumomab her family didn’t have any of these fears. They could be social, and both girls were able to attend school. It was a much more normal way of living. And with Gabby feeling well, she could participate in regular activities with the family.
In 2016, Gabby’s teacher noticed that she wasn’t seeing the board well. Her family took her to an optometrist and he noted catastrophic vision loss, as well as a mass behind her left eye. He incorrectly diagnosed it as retinoblastoma, but an eye biopsy at Sunnybrook in Toronto confirmed that it was the same genetic ALL for which Gabby was in treatment. Her central nervous system (CNS) and bone marrow testing both came back negative, and as such, her doctors identified it as an isolated ocular medullary relapse. Because of the relapse, Gabby was moved into a new protocol, but was still enrolled in the blinatumomab trial and being monitored.
Leukemia rarely forms into a tumourous cancer. Gabby’s case was extremely rare, and her doctors circulated her case around the world to get expert opinion on next steps. A decision was made to radiate the eye, followed by a bone marrow transplant at SickKids Hospital.
The tumour behind Gabby’s eye was completely dissolved with the radiation. There was no other viewable cancer shown by MRI. In December 2016, Gabby was moved to SickKids and she started chemotherapy treatment to prepare her for a bone marrow transplant. An unrelated umbilical cord donor was found that was a perfect match.
Gabby was given total body irradiation (TBI) as part of the treatment prior to her bone marrow transplant, and then went into isolation to prepare for her transplant. Her bone marrow transplant went extremely well. She grafted quickly and was discharged in early January 2017.
Because of the blinatumomab, Gabby went into her bone marrow transplant a very healthy kid. She did not experience any side effects from blinatumomab and her body responded well to the bone marrow transplant. She has been off all medication since May 2017 and has had no complicating factors.
Being in remission means that Gabby can focus on being a kid and participating in her favourite activities like reading, rock climbing, baking, and fashion. Like most kids her age, she loves online video games like Minecraft and Sims. She is an empathetic kid and is quick to stand up for herself or others against injustice.
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