Efficacy, Safety, and Dosing Protocols of Intracystic Bleomycin for Cystic Craniopharyngioma: A Scoping Review of Case Reports and Case Series

Abstract Background: Craniopharyngiomas are rare, benign, yet locally aggressive tumors of the sellar region. Their cystic nature and proximity to critical neuroendocrine structures complicate complete surgical excision and increase recurrence risk. Consequently, intracystic bleomycin (ICB) serves as an alternative therapy when safe surgical excision is unachievable. Materials and Methods: A scoping review was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews guidelines. Electronic databases including PubMed, Scopus, Cochrane, and ScienceDirect were searched. Case reports, series, and retrospective cohorts describing ICB dosing, safety, and efficacy were included. Data extraction covered patient demographics, tumor morphology, dosing regimens, leakage verification, and clinical outcomes. Results: Eighteen studies were included, spanning diverse geographic regions. Populations ranged from infants to elderly adults, with a predominance of pediatric cases. Administration protocols varied widely, with cumulative doses between 15 and 150 mg. Outcomes demonstrated significant cyst regression, with reductions exceeding 90% in some cases and sustained clinical improvement in up to 73% of patients. Adverse events included catheter-related complications, leakage, and neurotoxicity, occasionally resulting in hypothalamic injury or fatal cerebral edema. Gadolinium-enhanced magnetic resonance imaging proved superior for leakage detection and monitoring. Conclusion: ICB offers meaningful tumor control and reduced morbidity compared with aggressive surgery, particularly in pediatric populations. However, heterogeneity in dosing strategies and adverse event profiles underscores the need for standardized protocols. Future directions emphasize national registries, improved diagnostic monitoring, and prospective evaluation of long-term outcomes. Overall, ICB represents a valuable adjunct in multimodal management of cystic craniopharyngiomas, balancing efficacy with safety.

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Journal
Journal of Pediatric Neurosciences
Published
2026-10-27
DOI
https://doi.org/10.4103/jpn.jpn_83_26
Primary Topic
Pituitary Gland Disorders and Treatments
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article
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article

Efficacy, Safety, and Dosing Protocols of Intracystic Bleomycin for Cystic Craniopharyngioma: A Scoping Review of Case Reports and Case Series

Ved Prakash Maurya, Pradeep Chouksey, Daulat Singh, Swarjith Nimmakayala et al.
Journal of Pediatric Neurosciences
Pituitary Gland Disorders and Treatments
article

Efficacy, Safety, and Dosing Protocols of Intracystic Bleomycin for Cystic Craniopharyngioma: A Scoping Review of Case Reports and Case Series

Ved Prakash Maurya, Pradeep Chouksey, Daulat Singh, Swarjith Nimmakayala, Manish Gupta, Amit Agrawal, Sanjib Kumar Mishra
article en

Abstract

Abstract Background: Craniopharyngiomas are rare, benign, yet locally aggressive tumors of the sellar region. Their cystic nature and proximity to critical neuroendocrine structures complicate complete surgical excision and increase recurrence risk. Consequently, intracystic bleomycin (ICB) serves as an alternative therapy when safe surgical excision is unachievable. Materials and Methods: A scoping review was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews guidelines. Electronic databases including PubMed, Scopus, Cochrane, and ScienceDirect were searched. Case reports, series, and retrospective cohorts describing ICB dosing, safety, and efficacy were included. Data extraction covered patient demographics, tumor morphology, dosing regimens, leakage verification, and clinical outcomes. Results: Eighteen studies were included, spanning diverse geographic regions. Populations ranged from infants to elderly adults, with a predominance of pediatric cases. Administration protocols varied widely, with cumulative doses between 15 and 150 mg. Outcomes demonstrated significant cyst regression, with reductions exceeding 90% in some cases and sustained clinical improvement in up to 73% of patients. Adverse events included catheter-related complications, leakage, and neurotoxicity, occasionally resulting in hypothalamic injury or fatal cerebral edema. Gadolinium-enhanced magnetic resonance imaging proved superior for leakage detection and monitoring. Conclusion: ICB offers meaningful tumor control and reduced morbidity compared with aggressive surgery, particularly in pediatric populations. However, heterogeneity in dosing strategies and adverse event profiles underscores the need for standardized protocols. Future directions emphasize national registries, improved diagnostic monitoring, and prospective evaluation of long-term outcomes. Overall, ICB represents a valuable adjunct in multimodal management of cystic craniopharyngiomas, balancing efficacy with safety.

Journal of Pediatric Neurosciences
Sanjay Gandhi Post Graduate Institute of Medical Sciences (IN), All India Institute of Medical Sciences Bhopal (IN), Government Doon Medical College (IN), Utkal University (IN)
Good health and well-being
Openalex Percentile: Top 11%
Pituitary Gland Disorders and Treatments
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