Vinblastine and Methotrexate in Children With Pulmonary Vein Stenosis

• Status: Completed
• Trial end date: 2006-10-01
• Target enrollment: 0
• Participant gender: All

Summary

To evaluate the efficacy of the chemotherapeutic agents vinblastine and methotrexate in the treatment of two groups of children with multivessel pulmonary vein stenosis. Group 1 will contain children with multivessel pulmonary vein stenosis who do not have structural heart disease, and Group 2 will consist of children with multivessel pulmonary vein stenosis and concomitant structural heart disease.

The primary outcome variable for efficacy is patient status one year after the start of treatment, where status is classified as either failure or success. Failure is defined as death or evidence of progressive obstruction at any time over the course of treatment as defined in the protocol. Success constitutes complete or partial response to treatment or stability of disease. Secondary outcome variables for efficacy are survival, time from diagnosis of pulmonary vein stenosis until failure, and change in patient classification on a scale measuring the severity of the obstructive disease.

1.2 To assess the safety of vinblastine and methotrexate in the treatment of multivessel pulmonary vein stenosis.

The primary outcome variable for safety is any occurrence of toxicity related to the administration of the chemotherapeutic agents over the treatment period.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Boston Children's Hospital; Boston Children’s Hospital
• Collaborator: Dana-Farber Cancer Institute
• Treatments: Methotrexate; Vinblastine

Criteria

Inclusion Criteria:

• Diagnosis can be based on clinical and radiographic grounds or at the time of biopsy or prior surgical procedures. The diagnosis must be consistent with multivessel pulmonary stenosis.

• There must be evidence of severe pulmonary vein stenosis in at least two pulmonary veins.

• Evidence of myofibroblast neo-proliferation, if biopsies were obtained.

• Staging must include a complete cardiovascular evaluation including echocardiogram, and EKG.

• Accepted organ function includes:

• Creatinine < 1.5 x normal for age.

• SGPT, Bilirubin < 1.5 x normal for age.

• ANC ³ 1,500/mm3, Hemoglobin ³ 10g/dl, Platelets ³ 100,000/mm3

• Placement of a permanent central venous line. CVL access is necessary in all patients as vinblastine is a vesicant and will cause a tissue burn if infiltrated into the skin. Because a CVL may interfere with proper cardiac assessment in this patient population, the study physician will be made aware prior to the placement of a line.

• Patients may be listed for a lung transplant while enrolled on this study.

• All patients must have given written informed consent according to institutional guidelines.

Exclusion Criteria: