Sirolimus for Kaposiform Hemangioendothelioma and Kasabach-Merritt Phenomenon in a Neonate

Cabrera, Trevor B.; Speer, Allison L.; Greives, Matthew R.; Goff, Donna A.; Menon, Neethu; Reynolds, E. W.

doi:10.1055/s-0040-1718901

Cited by 9 publications

(6 citation statements)

References 30 publications

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“…Once infection symptoms occur during sirolimus treatment, it is necessary to actively treat them to avoid severe consequences. To reduce the side effects of sirolimus, some researchers have attempted to reduce its dosage [ 10 , 18 , 19 ]; the therapeutic dose was reduced to half the standard dose and even lower, and very good results were obtained. Among the cases in the present study, the standard dose (0.8 mg/L, bid) was used for all infants.…”

Section: Discussionmentioning

confidence: 99%

Efficacy of transcatheter arterial chemoembolization combined with sirolimus for treating Kasabach–Merritt phenomenon in infants, a retrospective study

Yin

Wang

et al. 2023

Annals of Medicine

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Objective This retrospective study aimed to observe the efficacy of transcatheter arterial chemoembolization (TACE) combined with sirolimus in the treatment of haemangioma combined with the Kasabach–Merritt phenomenon (KMP). Methods A total of 11 infants with KMP who were treated at our hospital from January 2016 to September 2021 were selected and treated with arteriosclerosis embolotherapy using a microsphere emulsion formed by bleomycin + ultra-fluid lipiodol + dexamethasone + contrast agent or bleomycin mixed microspheres as the embolising agent. The patients were administered sirolimus orally after TACE. The clinical efficacy and examination indicators before and after treatment were observed and compared. Results The 11 infants underwent TACE treatment by arteriosclerosis embolotherapy a total of 21 times; of these cases, 10 were cured, and 1 showed a moderate response. There were no cases of non-response or death. The platelet count rose from 10.0 (7.0, 18.0) x 10 9 /L before TACE to 236.0 (188.0, 275.0) x 10 9 /L six months after the first TACE, and the tumour size decreased from 49.0 (43.0, 111.7) cm 3 before TACE to 7.0 (3.5, 17.0) cm 3 six months after the first TACE. The differences were statistically significant (the Z values were −2.943 and −2.934, respectively, p < 0.05). Conclusion The combination of TACE and sirolimus has significant efficacy on critical children with KMP.

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Section: Discussionmentioning

confidence: 99%

Efficacy of transcatheter arterial chemoembolization combined with sirolimus for treating Kasabach–Merritt phenomenon in infants, a retrospective study

Yin

Wang

et al. 2023

Annals of Medicine

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“…Multiple treatment regimens have been used for KHE with KMP with varying success, and the most recent randomized clinical trials reported encouraging results of using combination therapy with sirolimus plus prednisolone ( 5 , 12 , 15 ). Sirolimus, an inhibitor of the mammalian target of rapamycin (mTor), has been demonstrated to show satisfactory efficacy as oral administration in the treatment of KHE ( 16 – 19 ). There are a number of published reports on the use of sirolimus monotherapy in KHE with KMP, and it appears to be effective and safe in patients with life-threatening vascular anomalies ( 18 , 20 ).…”

Section: Discussionmentioning

confidence: 99%

Case report: Kaposi hemangioendothelioma of the right upper limb with the Kasabach–Merritt phenomenon: A potentially lethal diagnostic challenge

Belani

Sapuan²,

Abdullah³

et al. 2022

Front. Pediatr.

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Kaposi hemangioendothelioma (KHE) is a rare vascular neoplasm that presents usually within the first year of life. Because of its rarity and complexity, there is often a delay in diagnosis. KHE could be associated with a life-threatening consumptive coagulopathy named the Kasabach–Merritt phenomenon (KMP). Here, we present the case of a 2-month-old girl who presented with progressive redness and swelling of her right upper limb over 6 weeks. Multiple health practitioners misdiagnosed her condition as an insect bite, cellulitis, and necrotizing fasciitis and gave treatment accordingly, which proved futile. A full blood count revealed bicytopenia of anemia and thrombocytopenia, a normal coagulation cascade, low fibrinogen, and raised D-Dimer levels. The imaging was suggestive of a high-flow vascular tumor likely to be a KHE. Subsequently, she was started on single-agent oral sirolimus with a dose increment to achieve satisfactory therapeutic levels and was treated for 1 year. She successfully completed the treatment regimen and had only transient hypertriglyceridemia, which resolved upon the completion of treatment. Currently, she is in remission 3 years after treatment. Keeping her case as an example, we would like to highlight the potentially lethal misdiagnosis of KHE with KMP, the importance of an early diagnosis of this condition, and the successful treatment outcome with single-agent sirolimus.

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“…To find a more suitable dose for neonates, we review the literature related to sirolimus used in neonates with other disorders. One case report suggested a dose of 0.4 mg/m 2 twice a day based on the goal levels clinically[ 11 ]. One study showed the proposed dose regimens for each age group, such as 0.4 mg/m 2 /dose twice a day for 0-1 mo, 0.5 for 1-2 mo, 0.6 for 2-3 mo, and so on, which was based on the developmental changes in pharmacokinetics[ 12 ].…”

Section: Discussionmentioning

confidence: 99%

Sirolimus treatment for neonate with blue rubber bleb nevus syndrome: A case report

Yang¹,

Yang²,

Yue³

et al. 2021

WJCC

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BACKGROUND Blue rubber bleb naevus syndrome (BRBNS) is a rare disease that usually presents with multiple venous malformations in the skin and gastrointestinal tract. Lesions located in the gastrointestinal tract always result in chronic gastrointestinal bleeding and severe anemia. The successful management of BRBNS with sirolimus had been reported in many institutions, due to its impact on signaling pathways of angiogenesis. However, the experience in treatment of neonates with BRBNS was limited. CASE SUMMARY A 38-day-old premature female infant born with multiple skin lesions, presented to our center complaining of severe anemia and hematochezia. Laboratory examination demonstrated that hemoglobin was 5.3 g/dL and contrast-enhanced abdominal computed tomography showed multiple low-density space-occupying lesions in the right lobe of the liver. She was diagnosed as having BRBNS based on typical clinical and examination findings. The patient was treated by transfusions twice and hemostatic drugs but symptoms of anemia were difficult to alleviate. A review of BRBNS case reports found that patients had been successfully treated with sirolimus. Then the patient was treated with sirolimus at an average dose of 0.95 mg/m 2 /d with a target drug level of 10-15 ng/mL. During 28 mo of treatment, the lesion was reduced, hemoglobin returned to normal, and there were no adverse drug reactions. CONCLUSION This case highlights the dosing regimen and plasma concentration in neonates, for the current common empiric dose is high.

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Sirolimus for Kaposiform Hemangioendothelioma and Kasabach-Merritt Phenomenon in a Neonate

Cited by 9 publications

References 30 publications

Efficacy of transcatheter arterial chemoembolization combined with sirolimus for treating Kasabach–Merritt phenomenon in infants, a retrospective study

Efficacy of transcatheter arterial chemoembolization combined with sirolimus for treating Kasabach–Merritt phenomenon in infants, a retrospective study

Case report: Kaposi hemangioendothelioma of the right upper limb with the Kasabach–Merritt phenomenon: A potentially lethal diagnostic challenge

Sirolimus treatment for neonate with blue rubber bleb nevus syndrome: A case report

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