Neoplasms

All Conditions

Hemangioma

Neoplasms, Vascular Tissue

Neoplasms by Histologic Type

Anti-Arrhythmia Agents

Antihypertensive Agents

Vasodilator Agents

All Drugs and Chemicals

Anti-Inflammatory Agents

Antineoplastic Agents

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Propranolol

Prednisolone

Methylprednisolone

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Glucocorticoids

Hormones

Hormone Antagonists

Antineoplastic Agents, Hormonal

Adrenergic beta-Antagonists

Adrenergic Agents

Adrenergic Antagonists

Neurotransmitter Agents

Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas

Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.

propranolol 0.5 mg/kg orally, 4 per day - 4-6 months

propranolol

Nancy M Bauman, MD

Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment. Others require treatment because they cause significant symptoms such as pain, or difficulty with breathing, eating or ambulating. Steroids have classically been used to treat hemangiomas and help to shrink them in 1/3 - 2/3 of patients. Unfortunately, steroids have many side effects in babies so physicians have sought other ways to treat them. Recently, the use of propranolol, a heart medication, was serendipitously found to reduce the size of hemangiomas. It appears to have many fewer side effects than steroids but it is not yet known if it works as well as steroids. This study seeks to compare the effect and the side effects of propranolol versus steroids for treating hemangiomas that cause symptoms in infants.

Infants with symptomatic hemangiomas will be enrolled. Magnetic resonance imaging will be completed before starting medication if the extent of the hemangioma is not evident on clinical examination alone. Infants will be randomized to receive either propranolol or steroids for 4-6 months. Hemangioma response will be measured and compared monthly as will tolerability of the medications. Additionally, urine specimens will be collected at each visit to determine if markers are present that can predict response to therapy.

Additionally, any hemangiomas that are excised will be examined for genetic markers to aid in predicting response to therapy.

Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas

Propranolol vs Prednisolone for Infant Hemangiomas-A Clinical and Molecular Study

A priori primary outcome was proportional change in the total surface area as measured by lesion's outer margin length x width at baseline minus the same measure at 4 months with surrogate data used at 5 months if 4 months not available.

All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular.

Number of serious adverse events experienced by the participants in each treatment arm within the categories adrenal crisis, growth/development, constitutional. Serious adverse events are defined as events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity, or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.

Number of Growth and Development AEs in each study arm

Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm

Number of allergy/immunology AE per study arm

Number of Dermatologic Adverse Events in each study arm.

Number of Endocrinologic AEs (of which adrenal crisis does not overlap).

Number of Gastrointestinal AEs in each arm

Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever)

Number of Metabolic or Laboratory AEs in each study arm.

Number of Vascular AEs in each study arm.

Number of constitutional AEs in each study arm.

Nancy Bauman

Professor, George Washington University, Attending Children;s National

Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas

propranolol: propranolol 0.5 mg/kg p.o. QID 6 months or less

Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.

Prednisolone: 1.0 mg/kg p.o. BID x 6 months or less

Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas

Propranolol 0.67 mg/kg p.o. TID x 4 - 6 months (2.0 mg/kg/day)

Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.

Prednisolone: 1.0 mg/kg p.o. BID x 4-6 months (2.0 mg/kg/day)

Total

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

Ethnicity (NIH/OMB)

United States

Region of Enrollment

Total surface area (length x width)

Adjusted total surface area (length x width x proportion of skin involved

Ulceration

Local

Segmental

Intermediate

Morphology

superficial

deep

mixed

Depth

Infants 2 weeks - 6 months of age with symptomatic, proliferating, infantile hemangiomas

Not every enrolled participant had an adverse event and some had more than one. The prednioslone participants who had adverse events warranting early withdrawal had severe failure to thrive (\<5th percentile), a serious adverse event.

Nancy M. Bauman MD, Principle Investigator

A priori analysis, TSA (length x width) at baseline versus at 4 months with surrogate data at 5 months Propranolol 0.67 mg/kg p.o. TID x 4 - 6 months (2.0 mg/kg/day)

A priori analysis, TSA (length x width) at baseline versus at 4 months with surrogate data at 5 months

Prednisolone: 1.0 mg/kg p.o. BID x 4-6 months (2.0 mg/kg/day)

Data available at 4 or 5 months for only 9/11 propranolol participants and for 6/8 prednisolone participants due to missed appointments. Overall, 90% (138/154) study appointments were completed.

Decrease in Size of Hemangioma (Length x Width) in Square mm

Adverse Events

Serious Adverse Events

All (total) study adverse events displayed as "Adverse events" including mild, moderate, and severe; as well as "Serious Adverse Events"

Overall Number of Adverse Events in Propranolol

Overall Number of Adverse Events in Prednisolone

All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular. In this table "Adverse Events" are those exclusive of the "Serious Adverse Events" which are noted separately.

Tolerability of Medication

Serious adverse events in propranolol treated participants.

Number of Serious Adverse Events in Propranolol

Serious adverse events in prednisolone treated participants

Number of Serious Adverse Events in Prednisolone

Number of Serious Adverse Events (SAEs)

Number of participants experiencing Growth/Development AEs

Growth/Developoment AEs Propranolol

Growth/Development AEs Prednisolone

Growth and Development Adverse Events

Number of participants experiencing pulmonary/respiratory AEs

Pulmonary/Respiratory AEs Propranolol

Pulmonary/Respiratory AEs Prednisolone

Pulmonary/Respiratory Adverse Events

Adverse events in allergy/immunology in propranolol treated participants

Allergy/Immunology Events Propranolol

Adverse events in allergy/immunology in prednisolone treated participants

Allergy/Immunology Events Prednisolone

Allergy/Immunology Adverse Events

Number of participants experiencing Dermatologic AEs

Dermatologic AEs Propranolol

Dermatologic AEs Prednisolone

Dermatologic Adverse Events

Number of participants experiencing Endocrinologic AEs.

Endocrine AEs Propranolol

Endocrinologic AEs Prednisolone

Endocrinologic Adverse Events

Number of Participants experiencing Gastrointestinal AEs

Gastrointestinal AEs Propranolol

Gastrointestinal AEs Prednisolone

Gastrointestinal Adverse Events

Number of participants experiencing Infectious AEs

Infectious AEs Propranolol

Infectious AEs Prednisolone

Infectious Adverse Events

Number of participants experiencing Metabolic or Laboratory AEs.

Metabolic/Laboratory AEs Propranolol

Number of participants experiencing Metabolic or Laboratory AEs in each study arm.

Metabolic/Laboratory AEs Prednisolone

Metabolic or Laboratory AEs

Number of participants experiencing Vascular AEs

Vascular AEs Propranolol

Vascular AEs Prednisolone

Vascular Adverse Events

Number of Participants experiencing Constitutional AEs

Constitutional AEs Propranolol

Constitutional AEs Prednisolone

Constitutional Adverse Events

Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas

propranolol: propranolol 0.67 mg/kg p.o. TID 4-6 months

Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.

Prednisolone: 1.0 mg/kg p.o. BID 4-6 months

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas	NCT00967226	Hemangioma of I...	propranolol Prednisolone	2 Weeks - 6 Months	Children's National Research Institute
Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas	NCT00967226	Hemangioma of I...	propranolol Prednisolone	2 Weeks - 6 Months	Children's National Research Institute
Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas	NCT00967226	Hemangioma of I...	propranolol Prednisolone	2 Weeks - 6 Months	Children's National Research Institute

Spots Global Cancer Trial Database for hemangioma of infancy