Acetylsalicylic acid suppression of the PI3K pathway as a novel medical therapy for head and neck lymphatic malformations

Bonilla‐Velez, Juliana; Whitlock, Kathryn B.; Ganti, Sheila; Zenner, Kaitlyn; Cheng, Chi Vicky; Jensen, Dana M.; Pham, Minh; Mitchell, Ryan M.; Dobyns, William B.; Bly, Randall A.; Bennett, James T.; Dahl, John P.; Perkins, Jonathan A.

doi:10.1016/j.ijporl.2021.110869

Cited by 7 publications

(2 citation statements)

References 47 publications

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“…Options include acetylsalicylic acid, sirolimus, or targeted therapies available through compassionate use protocols. 18,23,24,27 If the HNLM persists and medical therapy is not an option, IT is offered. IT can also be considered in patients who have failed medical management or at parental preference.…”

Section: Discussionmentioning

confidence: 99%

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Delaying Invasive Treatment in Unilateral Head and Neck Lymphatic Malformation Improves Outcomes

et al. 2022

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Objectives: Large (De Serres stage [IV-V]) head and neck lymphatic malformations (HNLMs) often have multiple, high-risk, invasive treatments (ITs) to address functional compromise. Logically reducing HNLM ITs should reduce treatment risk. We tested whether delaying HNLM ITs reduces total IT number.Materials: Consecutive HNLM patients (n = 199) between 2010 and 2017, aged 0-18 years. Methods: ITs (surgery or sclerotherapy) were offered for persistent or dysfunction causing HNLMs. Treatment effectiveness categorized by IT number: optimal (0-1), acceptable (2-5), or suboptimal (>5). Clinical data were summarized, and outcome associations tested (χ 2 ). Relative risk (RR) with a Poisson working model tested whether HNLM observation or IT delay (>6 months post-diagnosis) predicts treatment success (i.e., ≤1 IT).Results: Median age at HNLM diagnosis was 1.3 months (interquartile range [IQR] 0-45 m) with 107/199(54%) male. HNLM were stage I-III (174 [88%]), IV-V (25 [13%]). Initial treatment was observation (70 [35%]), invasive (129 [65%]). Treatment outcomes were optimal (137 [69%]), acceptable (36 [18%]), and suboptimal (26 [13%]). Suboptimal outcome associations: EXIT procedure, stage IV-V, oral location, and tracheotomy (p < 0.001). Stage I-III HNLMs were initially observed compared with stage I-III having ITs within 6 months of HNLM diagnosis, had a 82% lower relative treatment failure risk ([i.e., >1 IT], RR = 0.09, 95% CI 0.02-0.36, p < 0.001). Stage I-III HNLMs with non-delayed ITs had reduced treatment failure risk compared with IV-V (RR = 0.47, 95% CI 0.33-0.66, p < 0.001).Conclusion: Observation and delayed IT in stage I-III HNLM ("Grade 1") is safe and reduces IT (i.e., ≤1 IT). Stage IV-V HNLMs ("Grade 2") with early IT have a greater risk of multiple ITs.

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Section: Discussionmentioning

confidence: 99%

“…Determining HNLM genotype may guide selection of targeted medical therapy. Options include acetylsalicylic acid, sirolimus, or targeted therapies available through compassionate use protocols 18,23,24,27 . If the HNLM persists and medical therapy is not an option, IT is offered.…”

Section: Discussionmentioning

confidence: 99%

Delaying Invasive Treatment in Unilateral Head and Neck Lymphatic Malformation Improves Outcomes

et al. 2022

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Treatments and Outcomes of Pediatric Head and Neck Lymphatic Malformations: A 20‐Year Single Institution Experience

Zhao,

Thompson,

Weiss

et al. 2024

Otolaryngol.--head neck surg.

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ObjectiveExamine the management and outcomes of pediatric head and neck lymphatic malformations (HNLMs).Study DesignRetrospective case series.SettingTertiary academic hospital.MethodsPatients aged 18 years or younger treated for HNLMs at Johns Hopkins Hospital from 2000 to 2023 were identified. Demographics, clinical characteristics, and treatment courses were recorded from charts. Outcome measures included estimated clinical post‐treatment size reduction and complications. Fisher's exact test was performed for comparisons between treatments.ResultsWe identified 101 pediatric patients (50.5% female) with HNLMs. Fifty‐two received unimodal treatment, 11 received multimodal treatment, and 38 underwent active observation or no treatment. Of those who received treatment, 44% had their first treatment before 2 years of age. 54.5% of all patients received sclerotherapy. Sclerotherapy caused nearly complete or complete resolution in 52.2% (n = 24) of low‐stage (de Serres Stages 1‐3) HNLMs and complete resolution in 26.1%. Most (71.4%) high‐stage (de Serres Stages 4‐5) malformations demonstrated limited size reduction response to all treatments. Surgery had a higher all‐inclusive complication rate (25%) compared to sclerotherapy (17%). Multimodal treatment strategies were most often used to treat microcystic malformations of all stages, achieving a 42.9% nearly complete or complete resolution rate.ConclusionLow‐stage and macrocystic pediatric HNLMs respond well to treatment. Sclerotherapy had the lowest complication rate. High‐stage malformations had limited size reduction response to multimodal treatment. As new medical therapies continue to develop, opportunities for improved treatment algorithms will emerge.

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Alpelisib for the treatment of PIK3CA-related head and neck lymphatic malformations and overgrowth

Wenger¹,

Ganti²,

Bull³

et al. 2022

Genetics in Medicine

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Acetylsalicylic acid suppression of the PI3K pathway as a novel medical therapy for head and neck lymphatic malformations

Cited by 7 publications

References 47 publications

Delaying Invasive Treatment in Unilateral Head and Neck Lymphatic Malformation Improves Outcomes

Delaying Invasive Treatment in Unilateral Head and Neck Lymphatic Malformation Improves Outcomes

Treatments and Outcomes of Pediatric Head and Neck Lymphatic Malformations: A 20‐Year Single Institution Experience

Alpelisib for the treatment of PIK3CA-related head and neck lymphatic malformations and overgrowth

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