Intracystic bleomycin for cystic craniopharyngiomas in children

 

Abstract

Background

Craniopharyngiomas are the commonest benign histological tumours to involve the hypothalamo-pituitary region in childhood. Cystic craniopharyngiomas occur in more than 90% of tumours. The optimal treatment of cystic craniopharyngioma remains controversial. Radical resection is the treatment of choice in patients with favourable tumour localization. When the tumour localization is unfavourable, a gross-total or partial resection followed by radiotherapy is the main treatment option in adults. However, it presents risk of morbidity especially for children. Intracystic bleomycin has been utilized to potentially delay the use of radiotherapy or radical resection to decrease morbidity.

Objectives

To determine the benefits and harms of intracystic bleomycin versus other treatments for cystic craniopharyngiomas in children.

Search methods

We searched the electronic databases of CENTRAL (The Cochrane Library 2010, Issue 4), MEDLINE/PubMed (from 1966 to Oct 2010), and EMBASE/Ovid (from 1980 to Oct 2010) with pre-specified terms. In addition, we searched reference lists of relevant articles and reviews, conference proceedings and ongoing trial databases.

Selection criteria

Randomised controlled trials (RCTs) quasi-randomised trials or controlled clinical trials (CCTs) comparing intracystic bleomycin and other treatments for cystic craniopharyngiomas in children (from birth to 18 years).

Data collection and analysis

Two review authors independently performed the data extraction and the 'Risk of bias' assessment. We used risk ratio (RR) for binary data and mean difference (MD) for continuous data. We planned that if one of the treatment groups experienced no events and there was only one study available for the outcome, we would use the Fischer's exact test.

Main results

We could not identify any studies in which the only difference between the treatment groups was the use of intracystic bleomycin. We did identify a RCT comparing intracystic bleomycin with intracystic ³²P (n = 7 children). The trial had a high risk of bias. Survival could not be evaluated. There was no evidence of a significant difference in cyst reduction (MD = -0.15, 95% confidence interval (CI) -0.69 to 0.39, P= 0.59), neurological status (Fisher's exact P = 0.429), 3rd nerve paralysis (Fischer's exact P = 1.00), fever (RR = 2.92, 95% CI 0.73 to 11.70, P = 0.13) and total adverse effects (RR = 1.75, 95% CI 0.68 to 4.53, P = 0.25 ) between the treatment groups. There was a significant difference in favour of the ³²P group for the occurrence of headache and vomiting (Fischer's exact P = 0.029 for both outcomes).

Authors' conclusions

Since no RCTs, quasi-randomised trials or CCTs in which only the use of intracystic bleomycin differed between the treatment groups in the treatment of cystic craniopharyngiomas in children, no definitive conclusions could be made about the effects of intracystic bleomycin in these patients. Only one low-power RCT comparing intracystic bleomycin with intracystic ³²P treatment was available, but no definitive conclusions can be made about the effectiveness of these agents in children with cystic craniopharyngiomas. Based on the currently available evidence, we are not able to give recommendations for the use of intracystic bleomycin in the treatment of cystic craniopharyngiomas in children. High quality RCTs are needed.

 

Plain language summary

Intracystic bleomycin for children with cystic craniopharyngiomas

Cystic craniopharyngiomas are the commonest characteristic of craniopharyngiomas. Radical resection alone is not sufficient because the rate of recurrence is high and this procedure results in a high risk of endocrinological/neurological deficits. While in adults radiotherapy represents a valid postoperative adjunctive therapy, in children it has a high risk of side effects. Intracystic bleomycin have been used to potentially decrease side effects associated with cystic craniopharyngioma. This systematic review focused on (randomised) controlled studies. The authors could not identify any randomised controlled trials (RCTs) , quasi-randomised trials or controlled clinical trials (CCTs) in which the only difference between the intervention and control group was the use of intracystic bleomycin. They did identify one RCT comparing intracystic bleomycin with intracystic ³²P. The study is of low quality and the sample size is too small to detect the difference in outcome. Nowadays, the therapeutic use of intracystic bleomycin in children with cystic craniopharyngiomas remains uncertain. More high quality studies are needed.