Busulfan Treatment for Myeloproliferative Disease may Reduce Injection Burden in Vascular Endothelial Growth Factor-Driven Retinopathy

Purpose: Myeloproliferative neoplasms (MPNs) have been associated with increased systemic levels of vascular endothelial growth factor (VEGF). This study investigated an association between systemic busulfan for treatment of MPN and the requirement for intravitreal anti-VEGF injections for treatment of retinal pathology. Methods: Retrospective chart review of patients receiving systemic busulfan for myeloproliferative neoplasm and intravitreal anti-VEGF injections for macular and retinal vascular diseases from 2007 to 2021. Results: Of seven patients receiving oral busulfan for a hematological neoplasm and having concomitant retinal pathology requiring intravitreal anti-VEGF, all were white females >60 years old with MPN and exudative age-related macular degeneration. Of these, two patients had a reduced anti-VEGF requirement while on systemic busulfan, two took busulfan for fewer than 5 months, one developed retinal pathology over one year after stopping busulfan, one developed new retinal pathology while taking busulfan, and one had limited follow-up. Of the two patients with reduced anti-VEGF requirement while taking systemic busulfan, both had JAK2 V617F mutated MPN, and subsequent busulfan discontinuation was associated with an increased requirement for intravitreal anti-VEGF injections. Conclusions and importance: Systemic busulfan for treatment of MPN was associated with a reduced requirement for intravitreal anti-VEGF injections for retinal vascular disease in two patients. This association could be a result of inhibition of proliferative angiogenesis or reduced systemic VEGF levels with effective systemic treatment for MPN. Further study is required to confirm this association and determine whether this relationship is specific to busulfan or extends to other systemic medications used to treat MPN.