RARE DISEASE
Rare infantile hemangioma
Rare infantile hemangioma
Rare infantile hemangioma
Synonyms: Infantile hemangioma of rare localization
Synonyms: Infantile hemangioma of rare localization
Synonyms: Infantile hemangioma of rare localization
Drug discovery
1
drug
With orphan designation
Overview
Infantile hemangiomas (IHs) are benign vascular tumors affecting 4–10% of infants, typically emerging postnatally, proliferating rapidly, and involuting over years. While most resolve spontaneously, rare complex cases (e.g., segmental IH, PHACE syndrome, or lesions obstructing vital structures) require early intervention due to risks of ulceration, disfigurement, or functional impairment (e.g., vision, airway). First-line therapy involves oral propranolol, with multidisciplinary care for severe or systemic involvement [1][4][6][18].
Population
Affects 4–10% of infants, with higher prevalence in females (1.4:1–3:1 ratio), preterm infants, and those with low birth weight [2][6][7].
Segmental IHs and PHACE syndrome disproportionately affect females (9:1 ratio) [4][12].
Rising incidence linked to decreasing gestational age and birth weight [7][17].
Burden
10–24% develop complications (ulceration, bleeding, vision/airway obstruction) [4][6][16].
PHACE syndrome and internal IHs (e.g., liver, CNS) risk life-threatening outcomes (cardiac failure, hypothyroidism) [6][11][19].
Long-term sequelae include scarring, psychosocial impact, and rare systemic morbidity [4][16][19].
Therapies
First-line: Oral propranolol (2 mg/kg/day) for high-risk or complicated lesions [3][8][18].
Adjunctive: Topical timolol for superficial IHs; corticosteroids if beta-blockers are contraindicated [8][10].
Procedural: Pulsed-dye laser for ulceration/residual vessels; surgery for scarring, functional threats, or rebound growth [1][3][8].
Categories: rare circulatory system diseases, rare developmental anomalies during embryogenesis, rare neoplastic diseases
Research Papers
329 drug discovery papers about Rare infantile hemangioma, with 1 first-in-class and 11 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
329 drug discovery papers about Rare infantile hemangioma, with 1 first-in-class and 11 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-07-23 | Vascular Masquerade: A Case of Segmental Infantile Hemangioma With Minimal or Arrested Growth and Ulceration
Infantile hemangiomas (IHs) are the most common vascular tumors in pediatric patients. Infantile hemangiomas with minimal or arrested growth (IH-MAG) have a more atypical clinical appearance and are often misdiagnosed as capillary or other vascular malformations. Moreover, the histopathologic appearance of IH-MAG and ulcerated IH are not well characterized. A 5-month-old female presented with an asymptomatic blanchable vascular patch on the right thigh and lower leg and was diagnosed with a capillary malformation. At 8 months of age, the lesion developed spontaneous black eschars, concerning for a thrombo-occlusive process. Histologic examination revealed necrosis of the epidermis and superficial dermis overlying superficial dermal vessels with intravascular fibrinous thrombi and stained positive for glucose transporter protein-1. The patient was diagnosed with an IH-MAG and improved following initiation of propranolol therapy. This case highlights the ability of IH-MAG to masquerade as other vascular anomalies and offers rare insight into the pathophysiologic etiology of ulcerated IH.
2026-07-15 | Propranolol monotherapy versus propranolol plus prednisone for diffuse infantile hepatic haemangioma: a randomized clinical trial.
Oral propranolol, with or without corticosteroids, has been shown to benefit diffuse infantile hepatic haemangioma (IHH); randomized trial evidence is lacking. We aimed to evaluate the efficacy and safety of propranolol monotherapy versus propranolol plus prednisone for the treatment of problematic IHHs to determine whether oral prednisone provided an added benefit to propranolol. We conducted an open-label, multicenter, randomized controlled trial across six referral centers in China from July 2019 to November 2023 (clinicaltrials.gov registration: NCT03331744). Infants with problematic diffuse IHH were assigned (1:1) to oral propranolol alone or propranolol plus a short course of prednisone. The primary endpoint was the proportion of patients who achieved a lesion response at week 4 on serial ultrasound. Analyses followed the intention-to-treat principle. Forty-five patients were included in this study (propranolol, n = 22; propranolol plus prednisone, n = 23). Four weeks following treatment, lesion response rates were higher with propranolol plus prednisone than with propranolol alone [21/23 (91.3%) vs. 13/22 (59.1%); difference: 32.2%; 95% confidence interval (CI) = 6.9%-53.5%; P = 0.007]. Compared with monotherapy, combination therapy yielded higher lesion response rates during weeks 1-3, more rapid stabilization of thyroid-stimulating hormone levels during weeks 2-4, and a greater overall volumetric response at month 6 (91.3% vs. 63.6%; difference: 27.7%; 95% CI = 3.1%-49.3%; P = 0.026). Total adverse events were similar between the groups, and no grade 3-4 adverse events were observed. Addition of prednisone to propranolol in patients with diffuse IHH markedly improved lesion response rates. Propranolol plus prednisone represents a valid treatment for diffuse IHH.
2026-06-25 | Shuddering Attacks in an Infant Treated with Atenolol for Infantile Hemangioma: A Previously Unreported Adverse Effect
Complicated infantile hemangiomas require treatment, often with oral beta-blockers. Atenolol, a selective beta-blocker with rare neurological side effects, is considered a safer alternative to propranolol. This article reports a case of shuddering attacks, occurring during atenolol therapy, highlighting a previously unreported potential medication-related adverse effect.
2026-05-25 | Successful Treatment of Multiple Infantile Hemangiomas with Atenolol after Propranolol Failure
Impact of lactose; Powder Flowability and Capsu; Infantile hemangioma (IH) is the most common benign vascular tumor of childhood.In rare cases, it may occur as multiple cutaneous hemangiomas, which can be associated with visceral involvement and may require systemic therapy.Propranolol is currently considered the first-line treatment for IH; however, therapeutic resistance has occasionally been reported.We report the case of a 4-yearold girl presenting with ten multiple cutaneous infantile hemangiomas that had appeared since the age of one month.The patient had previously received oral propranolol at a dose of 3 mg/kg/day for one year without any clinical improvement.Owing to the persistence of the lesions and cosmetic concern, oral atenolol was initiated at a dose of 2 mg/kg/day after exclusion of visceral involvement by abdominal ultrasound.A marked regression of the subcutaneous component and significant fading of the lesions were observed after three months, with complete remission of the majority of lesions after six months.No adverse effects or recurrence were noted during follow-up.This case highlights atenolol as an effective and well-tolerated therapeutic alternative in patients with multiple infantile hemangiomas resistant to propranolol.
2026-05-22 | Propranolol in the Treatment of Infantile Hemangiomas: A Single-Center Experience.
Infantile hemangiomas (IH) are common benign vascular tumors of infancy that typically undergo gradual spontaneous regression over several years, although a minority require treatment. Propranolol, a non-selective β‑adrenergic antagonist, is the first-line therapy for complicated or high-risk IH, demonstrating both efficacy and a favorable safety profile. We retrospectively analyzed 37 infants treated with oral propranolol at a single center. Patients were monitored in-hospital during treatment initiation and at regular, outpatient visits. The response was assessed using clinical measurements, ultrasonography, and standardized photographs. Most patients had solitary lesions predominantly located in the head region. Complete regression occurred in 26 patients (70.3%) and partial regression in 11 (29.7%). Treatment duration ranged from 2 to 24 months (mean 10.1 months). Adverse events were rare and mild, including one case of hypoglycemia and one of transient somnolence. Oral propranolol is a safe and effective first-line therapy for IH, particularly when initiated early during the proliferative phase.
2026-07-23 | Vascular Masquerade: A Case of Segmental Infantile Hemangioma With Minimal or Arrested Growth and Ulceration
Infantile hemangiomas (IHs) are the most common vascular tumors in pediatric patients. Infantile hemangiomas with minimal or arrested growth (IH-MAG) have a more atypical clinical appearance and are often misdiagnosed as capillary or other vascular malformations. Moreover, the histopathologic appearance of IH-MAG and ulcerated IH are not well characterized. A 5-month-old female presented with an asymptomatic blanchable vascular patch on the right thigh and lower leg and was diagnosed with a capillary malformation. At 8 months of age, the lesion developed spontaneous black eschars, concerning for a thrombo-occlusive process. Histologic examination revealed necrosis of the epidermis and superficial dermis overlying superficial dermal vessels with intravascular fibrinous thrombi and stained positive for glucose transporter protein-1. The patient was diagnosed with an IH-MAG and improved following initiation of propranolol therapy. This case highlights the ability of IH-MAG to masquerade as other vascular anomalies and offers rare insight into the pathophysiologic etiology of ulcerated IH.
2026-07-15 | Propranolol monotherapy versus propranolol plus prednisone for diffuse infantile hepatic haemangioma: a randomized clinical trial.
Oral propranolol, with or without corticosteroids, has been shown to benefit diffuse infantile hepatic haemangioma (IHH); randomized trial evidence is lacking. We aimed to evaluate the efficacy and safety of propranolol monotherapy versus propranolol plus prednisone for the treatment of problematic IHHs to determine whether oral prednisone provided an added benefit to propranolol. We conducted an open-label, multicenter, randomized controlled trial across six referral centers in China from July 2019 to November 2023 (clinicaltrials.gov registration: NCT03331744). Infants with problematic diffuse IHH were assigned (1:1) to oral propranolol alone or propranolol plus a short course of prednisone. The primary endpoint was the proportion of patients who achieved a lesion response at week 4 on serial ultrasound. Analyses followed the intention-to-treat principle. Forty-five patients were included in this study (propranolol, n = 22; propranolol plus prednisone, n = 23). Four weeks following treatment, lesion response rates were higher with propranolol plus prednisone than with propranolol alone [21/23 (91.3%) vs. 13/22 (59.1%); difference: 32.2%; 95% confidence interval (CI) = 6.9%-53.5%; P = 0.007]. Compared with monotherapy, combination therapy yielded higher lesion response rates during weeks 1-3, more rapid stabilization of thyroid-stimulating hormone levels during weeks 2-4, and a greater overall volumetric response at month 6 (91.3% vs. 63.6%; difference: 27.7%; 95% CI = 3.1%-49.3%; P = 0.026). Total adverse events were similar between the groups, and no grade 3-4 adverse events were observed. Addition of prednisone to propranolol in patients with diffuse IHH markedly improved lesion response rates. Propranolol plus prednisone represents a valid treatment for diffuse IHH.
2026-06-25 | Shuddering Attacks in an Infant Treated with Atenolol for Infantile Hemangioma: A Previously Unreported Adverse Effect
Complicated infantile hemangiomas require treatment, often with oral beta-blockers. Atenolol, a selective beta-blocker with rare neurological side effects, is considered a safer alternative to propranolol. This article reports a case of shuddering attacks, occurring during atenolol therapy, highlighting a previously unreported potential medication-related adverse effect.
2026-05-25 | Successful Treatment of Multiple Infantile Hemangiomas with Atenolol after Propranolol Failure
Impact of lactose; Powder Flowability and Capsu; Infantile hemangioma (IH) is the most common benign vascular tumor of childhood.In rare cases, it may occur as multiple cutaneous hemangiomas, which can be associated with visceral involvement and may require systemic therapy.Propranolol is currently considered the first-line treatment for IH; however, therapeutic resistance has occasionally been reported.We report the case of a 4-yearold girl presenting with ten multiple cutaneous infantile hemangiomas that had appeared since the age of one month.The patient had previously received oral propranolol at a dose of 3 mg/kg/day for one year without any clinical improvement.Owing to the persistence of the lesions and cosmetic concern, oral atenolol was initiated at a dose of 2 mg/kg/day after exclusion of visceral involvement by abdominal ultrasound.A marked regression of the subcutaneous component and significant fading of the lesions were observed after three months, with complete remission of the majority of lesions after six months.No adverse effects or recurrence were noted during follow-up.This case highlights atenolol as an effective and well-tolerated therapeutic alternative in patients with multiple infantile hemangiomas resistant to propranolol.
2026-05-22 | Propranolol in the Treatment of Infantile Hemangiomas: A Single-Center Experience.
Infantile hemangiomas (IH) are common benign vascular tumors of infancy that typically undergo gradual spontaneous regression over several years, although a minority require treatment. Propranolol, a non-selective β‑adrenergic antagonist, is the first-line therapy for complicated or high-risk IH, demonstrating both efficacy and a favorable safety profile. We retrospectively analyzed 37 infants treated with oral propranolol at a single center. Patients were monitored in-hospital during treatment initiation and at regular, outpatient visits. The response was assessed using clinical measurements, ultrasonography, and standardized photographs. Most patients had solitary lesions predominantly located in the head region. Complete regression occurred in 26 patients (70.3%) and partial regression in 11 (29.7%). Treatment duration ranged from 2 to 24 months (mean 10.1 months). Adverse events were rare and mild, including one case of hypoglycemia and one of transient somnolence. Oral propranolol is a safe and effective first-line therapy for IH, particularly when initiated early during the proliferative phase.
Access all drug discovery papers and probability of success in trials forecasts:
Access all drug discovery papers and probability of success in trials forecasts:
Drug Discovery Landscape
1 orphan drug designation for Rare infantile hemangioma, including 1 approved therapy.
1 orphan drug designation for Rare infantile hemangioma, including 1 approved therapy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
propranolol [HEMANGEOL] | small molecules | FDA | 2008-09-05 | 2014-03-14 | Eton Pharmaceuticals, Inc. |
Let's accelerate rare disease drug discovery
Let's accelerate drug discovery
Get access to Explority AI's forecasts to outperform average preclinical success rates. Whether you're expanding your R&D pipeline, evaluating a partnership, or simply have a question — we'd love to hear from you.