Dr Gasparini on Challenges With Oral Therapies in Pediatric Patients or Patients With Dysphagia

“The biggest challenge with administering treatments to patients who have difficulty swallowing, whether [they are] a very young patient who hasn’t learned how to swallow pills or capsules or even our young adult patients with dysphasia, is whether these medications that the patients need are available in a liquid formulation.”

Kate Gasparini, PharmD, BCOP, BCPPS, a pediatric oncology clinical pharmacy specialist at Memorial Sloan Kettering Cancer Center, discussed potential issues with administering oral agents in pediatric patients with cancer or patients with dysphagia.

One of the primary challenges in administering oral treatments in patients with cancer who have trouble swallowing, whether they are pediatric or have dysphagia, is the availability of a liquid formulation of a given drug, Gasparini began. Some medications can be compounded into a liquid formulation, dissolved, or manipulated by the patient or their caregiver so that they can be given, she continued. Although some medications have predetermined instructions on how to compound or dissolve them, oftentimes these processes require research on how to do this safely, she added.

Other barriers to giving oral therapies include identifying a pharmacy that can perform compounding near where patients live, Gasparini said. There are also instances in which there are no available compounding recipes, she noted.

Moreover, safely compounding agents at home often requires a lot of education and supplies for the patient’s families or caregivers, which can often be cumbersome, she said. Patients’ parents and/or caregivers may also be worried about correctly performing this process and about making errors, Gasparini added.

These barriers to treatment underscore the need for formulations that can be orally administered in a safe manner. In November 2024, the FDA approved an oral solution of imatinib (Imkeldi) for the treatment of patients with certain forms of leukemia and other types of cancer. The regulatory decision included indications in pediatric patients with newly diagnosed Philadelphia chromosome (Ph)–positive acute lymphoblastic leukemia in combination with chemotherapy as well as adult and pediatric patients with Ph-positive chronic myeloid leukemia in chronic phase. The flavored solution offers patients a therapeutic option that can be administered precisely in order to improve treatment adherence and accessibility.