Expanding access to tyrosine kinase inhibitors for treatment of Philadelphia chromosome–positive acute lymphoblastic leukemia in low- and middle-income countries.

1600 Background: Acute lymphoblastic leukemia (ALL) is a hematological malignancy affecting 300,000 people worldwide, with an estimated incidence of 100,000 new cases annually. An important cytogenetic abnormality, the Philadelphia chromosome, causes an aggressive form of disease that affects 3-4% of pediatric and 20-30% of adult patients. Since the early 2000s, tyrosine kinase inhibitors (TKIs) have dramatically transformed the treatment of Philadelphia chromosome positive ALL (Ph+ALL). In high-income countries, different TKI regimens in combination with chemotherapy achieve remission rates >90%, but survival rates in low- and middle-income countries (LMICs) are persistently worse, largely due to access barriers, including high costs of innovative therapies. Methods: The Max Foundation provides TKIs at no cost to eligible patients with Ph+ALL and other diseases in resource-limited settings worldwide. We conducted a retrospective review of all patients with a diagnosis of PH+ALL initiating treatment with TKIs between January 1, 2007 and July 31, 2025 through the Glivec International Patient Assistance Program (2007-2017) or Max Access Solutions (since 2017). Data on age at diagnosis, treatments, sex, country of origin, program status, and reason for closure were analyzed using descriptive statistics, with correlations between available variables and length of treatment explored. Results: Of 1,671 patients enrolled, 342 (20.5%) remained active at the time of analysis. Males accounted for 967 patients (57.9%). Patients enrolled from 47 LMICs in the Americas (542, 32.4%), Asia excluding India (414, 24.8%), Eastern Europe (365, 21.8%), Africa (219, 13.1%), and India (131, 7.8%). Median age at diagnosis was 29, with 485 (29.0%) 18 years old or younger. Most patients (1594, 95.4%) initiated treatment with imatinib as first-line treatment; of these, 110 (6.9%) moved to a second-line treatment and 18 (1.1%) to a third-line. Among 1,329 closed cases, reasons for closure included death (526, 39.8%), lost to follow-up (291, 21.9%), and clinical reasons (260, 19.6%). Median treatment length was 17 months (range 0-197 months). Patients who died were older at diagnosis (median 34 vs 28 years old) and had a shorter median treatment duration (13 vs 19 months) compared with other patients. Treatment duration tended to decrease with age; with a significant negative correlation between age at diagnosis and total months of treatment ( r =-0.171, p<0.001). Since 2024, 25 patients received additional supportive services through The Max Foundation, including transportation assistance. Conclusions: This program demonstrates the feasibility of providing TKIs for the treatment of PH+ALL in diverse resource-limited geographies. Increasing access to TKIs is a critical step in eliminating global disparities in the burden of Ph+ALL.