Liquid Formulations Boost Treatment Adherence in Pediatric Patients With Cancer and Patients With Dysphagia

Kate Gasparini, PharmD, BCOP, BCPPS

Prior to the FDA approval of the liquid formulation of imatinib (Imkeldi), pediatric patients with cancer and those with dysphagia who required the medication faced several barriers to safely receiving the drug, according to Kate Gasparini, PharmD, BCOP, BCPPS.

“Whether or not a medication is available as a liquid formulation may seem trivial, but for those of us who work every day with this patient population, we know it makes all the difference,” Gasparini said in an interview with OncLive®. “It can truly improve the quality of life for patients and their families to have a liquid formulation that’s easy to administer and readily available for a patient who has difficulty swallowing, especially for oral chemotherapy agents, which are essential in the treatment of certain pediatric [patients with] cancer. It’s one less thing for our patients and families to worry about when they’re undergoing these intensive treatments and, as the treating team, we know the patient will be able to get the therapy safely and reliably. It’s an additional peace of mind.”

Gasparini is a pediatric oncology clinical pharmacy specialist at Memorial Sloan Kettering Cancer Center in New York, New York.

In November 2024, the FDA approved an oral solution of imatinib for the treatment of patients with certain forms of leukemia and other types of cancer.1 The agent was originally approved by the FDA in 2001 and is indicated for use in several cancer types including Philadelphia chromosome (Ph)–positive chronic myeloid leukemia (CML) in chronic phase, relapsed/refractory Ph-positive acute lymphoblastic leukemia (ALL), and myelodysplastic or myeloproliferative diseases associated with PDGFR gene rearrangements.2 It is also indicated for pediatric patients with Ph-positive CML in chronic phase and in combination with chemotherapy in newly diagnosed Ph-positive ALL.

In the interview, Gasparini discussed the challenges of administering oral therapies without a liquid formulation, the significance of the approval of the liquid solution of imatinib, and her role in counseling patients and families as they navigate treatment.

OncLive: How is a clinical pharmacist’s role in the treatment of patients with cancer who are unable to swallow pills?

Gasparini: I’m a clinical pharmacy specialist with 10 years of experience, specifically in pediatric oncology. In my current role, I rotate through inpatient service and 3 of our outpatient clinics. When I’m in the outpatient clinics, I conduct direct patient care visits. During those visits, I review medications, including chemotherapy agents, with patients and families, [and] I suggest modifications or adjustments to therapy in conjunction with the clinical team.

My role in the management of pediatric patients with cancer consists of a lot of supportive care and symptom management for patients [who are] actively receiving chemotherapy. In terms of treatment planning, I work very closely with the [clinical] team for patients starting new chemotherapy regimens or those requiring a modification in therapy to recommend appropriate dosing of various agents and supportive care medications. I [provide] education with patients and families on the administration of these medications in the home with adverse effect [AE] monitoring and counseling. I [also] assist the clinical team with medication procurement when necessary.

What are some of the challenges with administering oral treatments in pediatric patients or adult patients with difficulty swallowing?

The biggest challenge with administering treatments to patients who have difficulty swallowing, whether they are a young patient who hasn’t learned how to swallow pills or capsules yet, or our young adult patients with dysphagia, is whether the medications that the patients need are available in a liquid formulation. If there’s not a liquid formulation available, it is part of my role to help the team navigate whether the medications can be compounded into a liquid formulation for administration, or [if they can be] safely dissolved or manipulated by the patient or their families so that it can be given.

Depending on the medication, there could be established recipes for compounding or instructions on how to dissolve [it]. However, a lot of the time, there is some digging into the data and literature that is required to [determine] how to safely do this. Another barrier is identifying compounding pharmacies that patients can use to get medications made into a liquid formulation, if there is a compounding recipe available.

In the cases where we have patients or their families dissolve medications for administration at home, there’s a lot of education needed to do this safely. Depending on the medication, a lot of times they need to have supplies such as gowns, masks, gloves, medicine cups, syringes, pill crushers, and/or splitters. A lot goes into it.

This can be cumbersome for families. They might need to be educated several times. I’ve heard directly from caregivers and parents that they just want to do it correctly. I’ve had a dad say to me, ‘I just don’t want to mess this up.’ They just want to be able to administer it safely to their child.

What types of support do you provide for patients and their families for at-home formulations?

We have a lot of reminders and documentation that we give to patients on safely receiving oral chemotherapy at home. It can be overwhelming for families, so I always tell them that if they get home and forget what they’re doing, please call the clinic. I will go through this with a parent as many times as they need to feel safe in giving this to their child.

It can be very time consuming and cumbersome for patients and their families. It’s helpful to have a checklist of what they need to have in the home in order to administer [treatments] safely. In the beginning, [we provide] written instructions in plain language [explaining] methods for dissolving and discarding the medication. [We provide] things that supplement the in-person education that we are doing.

What are some considerations for selecting the liquid formulation of imatinib?

I was very excited to learn about the approval of [the liquid formulation] of imatinib. In my role, for as long as I’ve been doing it, I have educated patients and their families on either dissolving imatinib for use or helping them find a compounding pharmacy that could make the extemporaneously compounded oral suspension. It’s always going to be the preference of the team to use a dosage form that is easiest for patients and families to administer. For pediatric patients or those who are unable to swallow [pills], it will usually be the commercially available liquid formulation.

A potential barrier [to access of the liquid formulation] could be insurance coverage, as well as the time it may take to get all of the necessary approvals and getting them filled through a specialty pharmacy. Oftentimes, it may be urgent for patients to start this therapy. [We may need to] consider first starting the patient with dissolving tablets or compounded a suspension while we are working to obtain the [liquid] formulation.

It's great that it is available to patients because they’re on the therapy for a long time. It makes dose modifications easier, and [allows us] to administer it directly to the patients in a formulation that’s easy to give.

Are there any toxicities that are unique to liquid formulations?

In general, the liquid formulations have the same general AE profile [as other forms], but a key consideration with liquid formulations is volume. For our younger patients, or even our young adult patients with dysphagia, larger volumes can oftentimes be difficult to tolerate. Another concern is palatability and taste. For any liquid formulations that have a larger volume or don’t taste good, I worry about it potentially causing nausea, or there being any type of aversion that could lead to poor adherence.

References

1. Shorla Oncology announces FDA approval of Imkeldi (imatinib) oral solution, an oral liquid for the treatment of certain forms of leukemia and other cancers. News release. Shorla Oncology. November 25, 2024. Accessed June 18, 2025. https://www.businesswire.com/news/home/20241125044117/en
2. Imkeldi. Prescribing information. Shorla Oncology; 2001. Accessed June 18, 2025. https://shorlaoncology.com/pdf/Imkeldi_PrescribingInformation.pdf