Trial Outcomes & Findings for Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas (NCT NCT00555464)

Results Overview

Response of IH not confined to the dermis will be coded using the following criteria: Progressive disease: \>40% increase in volume by MRI, Partial response: \>65% reduction in volume by MRI, Complete response: no visual or radiographic evidence of disease, Stable disease: none of the above or \<40% increase or \<65% decrease in volume by MRI. Response of superficial IH will be coded using the following criteria (based on RECIST): Progressive disease: \>30% increase in IH size, Partial response: \>30% reduction in size, Complete response: no evidence of disease, Stable disease: none of the above. Our first 3 patients showed limits to using MRI volume to measure IH size/response to therapy. Unlike other solid tumors, the superficial distribution of some IH made getting volume by MRI difficult, resulting in smaller tumor estimation compared to clinical assessment. Based on these observations, we amended the protocol to report response based on RECIST criteria instead of change in IH volume.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

6 weeks

Results posted on

2013-06-11

Participant Flow

Study participants were recruited from the Pediatric Dermatology clinic at Children's Hospital of Wisconsin between. Study enrollment period extended from 2008-2009.

Subjects meeting eligibility criteria including diagnosis and lesional size criteria were enrolled in the study. Subjects who had received topical or intralesional corticosteroids were eligible following a 1 week wash-out period.

Participant milestones

Participant milestones
Measure	Vincristine Treatment Group Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas. Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.	Oral Steroid Treatment Group The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment. Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week
Overall Study STARTED	4	4
Overall Study COMPLETED	4	1
Overall Study NOT COMPLETED	0	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Vincristine Treatment Group n=4 Participants Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas. Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.	Oral Steroid Treatment Group n=4 Participants The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment. Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age Continuous	0.18 years STANDARD_DEVIATION 0.05 • n=5 Participants	0.11 years STANDARD_DEVIATION 0.05 • n=7 Participants	0.15 years STANDARD_DEVIATION 0.06 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants	4 participants n=7 Participants	8 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 weeks

Population: Per protocol, all participants were analyzed, no matter the treatment arm or treatment success.

Response of IH not confined to the dermis will be coded using the following criteria: Progressive disease: \>40% increase in volume by MRI, Partial response: \>65% reduction in volume by MRI, Complete response: no visual or radiographic evidence of disease, Stable disease: none of the above or \<40% increase or \<65% decrease in volume by MRI. Response of superficial IH will be coded using the following criteria (based on RECIST): Progressive disease: \>30% increase in IH size, Partial response: \>30% reduction in size, Complete response: no evidence of disease, Stable disease: none of the above. Our first 3 patients showed limits to using MRI volume to measure IH size/response to therapy. Unlike other solid tumors, the superficial distribution of some IH made getting volume by MRI difficult, resulting in smaller tumor estimation compared to clinical assessment. Based on these observations, we amended the protocol to report response based on RECIST criteria instead of change in IH volume.

Outcome measures

Outcome measures
Measure	Vincristine Treatment Group n=4 Participants Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas. Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.	Oral Steroid Treatment Group n=3 Participants The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment. Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week
Response of Hemangioma (IH) to Treatment Progressive Disease	0 participants	1 participants
Response of Hemangioma (IH) to Treatment Partial Response	2 participants	0 participants
Response of Hemangioma (IH) to Treatment Complete Response	0 participants	0 participants
Response of Hemangioma (IH) to Treatment Stable Disease	2 participants	2 participants

SECONDARY outcome

Timeframe: Initial visit, 2, 4, 6, 10 and 12 weeks of therapy

Adverse events were closely monitored and recorded at weekly visits during treatment period and for two years after treatment ceased. Laboratory values were taken every other week during the treatment period. Please see Adverse Events module for more details.

Outcome measures

Outcome measures
Measure	Vincristine Treatment Group n=4 Participants Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas. Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.	Oral Steroid Treatment Group n=4 Participants The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment. Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week
Toxicity to Medications Patients with Serious Adverse Events	0 participants	0 participants
Toxicity to Medications Patients with Other Adverse Events	3 participants	0 participants

Adverse Events

Vincristine Treatment Group

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Oral Steroid Treatment Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Vincristine Treatment Group n=4 participants at risk Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas. Vincristine : Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.	Oral Steroid Treatment Group n=4 participants at risk The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment. Prednisone : Prednisolone given at 3 mg/kg/day by mouth for 12 week
Gastrointestinal disorders Gastrointestinal: weight loss	25.0% 1/4 • Number of events 1	0.00% 0/4
Blood and lymphatic system disorders Anemia	75.0% 3/4 • Number of events 5	0.00% 0/4
Respiratory, thoracic and mediastinal disorders obstruction/stenosis of airway	25.0% 1/4 • Number of events 1	0.00% 0/4
Infections and infestations Infection with normal ANC	25.0% 1/4 • Number of events 1	0.00% 0/4
Skin and subcutaneous tissue disorders Rash/desquamation	25.0% 1/4 • Number of events 1	0.00% 0/4

Additional Information

Beth Drolet, MD

Medical College of Wisconsin

Phone: 414-955-2817

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place