Vincristine, a chemotherapy drug primarily used in pediatric cancer treatments like acute lymphoblastic leukemia and Wilms’ tumor, is known for neurotoxic side effects, including a rare but under-discussed manifestation—vincristine-induced ptosis. This systematic review (PROSPERO: CRD42024617946) analyzed data from three databases, identifying 379 articles. Of these, 28 articles encompassing 31 unique pediatric cases were included, with Turkey contributing the highest number of cases (9 cases, 29.03%). The median age was 3 years (IQR: 2 – 5.5), with 58.06% male (18 cases) and 41.93% female (13 cases). Ptosis appeared bilaterally in 61.29% (19 cases) and unilaterally in 38.71% (12 cases), showing a left-side predominance in unilateral cases. The median time to symptom onset after the last vincristine dose was 6 days (IQR: 2 – 12). Treatment protocols varied; 74.19% (23 cases) adjusted or discontinued vincristine, with 34.78% (8 cases) restarting after skipping doses. Pyridoxine with or without pyridostigmine was used in 70% (14 of 20 treated cases), and recovery was generally favorable, with symptoms resolving within 28 days (IQR: 22.75 – 42) in most cases. Mild residual ptosis was noted in 9.67% (3 cases). Our review shows the significant variability inherent to management approaches in this patient demographic and highlights the need for standardized documentation and treatment approaches, proposing “VINTOSIS-3” as a practical treatment protocol. Furthermore, the VICTORIA (Vincristine-InduCed pTOsis cRIteriA) Grading is introduced to standardize ptosis severity evaluation. We further emphasize the importance of early detection, vigilant monitoring, and tailored interventions to balance neurotoxicity management with chemotherapy efficacy. <Table Float="No" ID="Taba"> <tgroup cols="2"> <colspec align="left" colname="c1" colnum="1" /> <colspec align="left" colname="c2" colnum="2" /> <tbody> <row> <entry nameend="c2" namest="c1"> What is Known: • Vincristine, a widely used chemotherapeutic agent in pediatric oncology, is associated with toxic side effects, primarily peripheral neuropathy. • Ocular complications of vincristine, including ptosis, have been reported but remain under-recognized. </entry> </row> <row> <entry nameend="c2" namest="c1"> What is New: • Vincristine-induced ptosis is predominantly bilateral, but when unilateral, it shows a left-sided predominance and typically appears after a median of four doses. • The proposed VICTORIA Grading standardizes severity assessment, and the VINTOSIS-3 protocol provides a structured approach to management. </entry> </row> </tbody> </tgroup> </Table>

Vincristine-induced ptosis in pediatric patients: a systematic review and practice recommendations

期刊论文
发表日期：

2025年2月24日

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H. Shafeeq Ahmed,
Chinmayee J. Thrishulamurthy

摘要

Vincristine, a chemotherapy drug primarily used in pediatric cancer treatments like acute lymphoblastic leukemia and Wilms’ tumor, is known for neurotoxic side effects, including a rare but under-discussed manifestation—vincristine-induced ptosis. This systematic review (PROSPERO: CRD42024617946) analyzed data from three databases, identifying 379 articles. Of these, 28 articles encompassing 31 unique pediatric cases were included, with Turkey contributing the highest number of cases (9 cases, 29.03%). The median age was 3 years (IQR: 2 – 5.5), with 58.06% male (18 cases) and 41.93% female (13 cases). Ptosis appeared bilaterally in 61.29% (19 cases) and unilaterally in 38.71% (12 cases), showing a left-side predominance in unilateral cases. The median time to symptom onset after the last vincristine dose was 6 days (IQR: 2 – 12). Treatment protocols varied; 74.19% (23 cases) adjusted or discontinued vincristine, with 34.78% (8 cases) restarting after skipping doses. Pyridoxine with or without pyridostigmine was used in 70% (14 of 20 treated cases), and recovery was generally favorable, with symptoms resolving within 28 days (IQR: 22.75 – 42) in most cases. Mild residual ptosis was noted in 9.67% (3 cases). Our review shows the significant variability inherent to management approaches in this patient demographic and highlights the need for standardized documentation and treatment approaches, proposing “VINTOSIS-3” as a practical treatment protocol. Furthermore, the VICTORIA (Vincristine-InduCed pTOsis cRIteriA) Grading is introduced to standardize ptosis severity evaluation. We further emphasize the importance of early detection, vigilant monitoring, and tailored interventions to balance neurotoxicity management with chemotherapy efficacy.

What is Known:

• Vincristine, a widely used chemotherapeutic agent in pediatric oncology, is associated with toxic side effects, primarily peripheral neuropathy.

• Ocular complications of vincristine, including ptosis, have been reported but remain under-recognized.

What is New:

• Vincristine-induced ptosis is predominantly bilateral, but when unilateral, it shows a left-sided predominance and typically appears after a median of four doses.

• The proposed VICTORIA Grading standardizes severity assessment, and the VINTOSIS-3 protocol provides a structured approach to management.