乳幼児肝巨大血管腫

BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.

Background: An infantile hemangioma (IH) is a benign lesion that develops as a result of pathologically dysregulated proliferation of the endothelial cells of the capillaries, typically appearing within the first three years of life. The disease is found mostly on the skin and-less often-in the internal organs. Although IH generally has a good prognosis and may sometimes undergo spontaneous regression, certain types of IH with a specific form, localization, and size may lead to life-threatening conditions including impairment of vital functions. This determines the need for precise diagnostics and treatment. Case presentation: The paper presents a clinical observation of an infant in the first months of life with IH presenting as diffuse hepatic hemangiomatosis and a hemangioma of the left eyebrow area. The IH was associated with life-threatening conditions. However, effective treatment with the non-selective β-adrenoblocker propranolol led to a positive outcome and the resolution of complications. Conclusions: This case demonstrates the critical importance of timely diagnosis and treatment for lesions that, while benign, can lead to fatal complications if not addressed promptly.

Infantile hepatic hemangiomatosis (IHH) is a rare benign vascular tumor of infancy, defined by multiple angiomatous lesions diffusely replacing the hepatic parenchyma. Its clinical course can be severe due to systemic complications, particularly consumptive hypothyroidism and high-output heart failure. We report the case of a four-month-old female infant admitted for abdominal distension evolving over two weeks. Physical examination revealed massive hepatomegaly (hepatic span of 14 cm) with abdominal-thoracic collateral venous circulation and no cutaneous hemangiomas. Laboratory workup showed normochromic normocytic non-regenerative anemia (hemoglobin = 6.7 g/dL), hepatic cytolysis (aspartate aminotransferase = 3× normal), cholestasis (alkaline phosphatase = 188 IU/L, gamma-glutamyl transferase = 335 IU/L), and a prothrombin time reduced to 54%. Alpha-fetoprotein was 470 ng/L. Abdominal MRI revealed multiple rounded hepatic lesions, T1 hypointense and T2 hyperintense with diffusion restriction and progressive centripetal contrast enhancement, consistent with diffuse IHH. Cardiac (echocardiography) and cerebral (transfontanellar ultrasound) assessments were normal. Systematic thyroid function tests revealed severe consumptive hypothyroidism, with thyroid-stimulating hormone level of 500 µIU/mL and free T4 of 6 pmol/L, without antithyroid antibodies. Management included progressive propranolol (Hemangiol®) up to 3 mg/kg/day, levothyroxine at 20 µg/kg/day, and red blood cell transfusion. At the six-month follow-up, thyroid function had normalized, and hepatic lesions showed significant regression. This case highlights the necessity of systematic thyroid function screening in all infants with IHH and illustrates the efficacy of propranolol in managing this rare but potentially life-threatening condition.

BACKGROUND Infantile hemangiomas (IH) are the most common benign vascular tumors in infants. While most of these lesions undergo spontaneous involution or respond favorably to propranolol, some cases present with early ulceration, aggressive progression, or resistance to standard therapy. Managing such complex cases remains challenging and requires individualized, multidisciplinary strategies and timely escalation to alternative modalities. Here, we report a medically refractory case of ulcerated infantile hemangioma and highlight the role of bleomycin sclerotherapy as a rescue therapy option. CASE REPORT We report the case of a 6-month-old female with multiple cutaneous and hepatic infantile hemangiomas, including a facial lesion with extensive ulceration involving the nasal and upper-lip regions. Histopathologic examination with positive GLUT-1 immunostaining confirmed the diagnosis of infantile hemangioma. Despite treatment with propranolol and adjunctive systemic corticosteroids, the ulceration worsened. Sirolimus was initiated but clinical deterioration continued within days, leading to the decision to provide interventional management. A single session of fluoroscopy-guided percutaneous bleomycin sclerotherapy was performed, resulting in rapid ulcer healing and sustained lesion stability. No adverse events occurred during or after the procedure. CONCLUSIONS Bleomycin sclerotherapy may be an effective and safe therapeutic option for propranolol resistance ulcerated infantile hemangiomas that fail conventional medical therapy. Early multidisciplinary evaluation and timely escalation to interventional treatment may help prevent progressive tissue destruction and improve functional and cosmetic outcomes in selected refractory cases.

Background: Infantile hepatic hemangiomas (IHH) are common benign vascular tumors in infancy with diverse presentations. Methods: We report a 7-week-old infant presenting with hepatosplenomegaly, multiple skin and hepatic hemangiomas, anemia, and recurrent lung infections. Results: Treatment included propranolol, corticosteroids, and sirolimus, along with antifungal prophylaxis with fluconazole. The patient developed pneumothorax and pulmonary aspergillosis. Despite antifungal therapy with voriconazole and liposomal amphotericin B, along with surgical intervention, her condition deteriorated, resulting in multi-organ failure and death at 8.5 months of age. Conclusions: This case illustrates the complexity of IHH management and highlights the risk of severe infections during immunosuppressive therapy even when standard prophylaxis protocols are applied.