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High Responsiveness of Head and Neck Kaposiform Hemangioendothelioma Without Kasabach-Merritt Phenomenon to Sirolimus Monotherapy.

Xu Y, Zhou J, Lan Y et al. · 2026 · Pediatric Blood & Cancer · Atlas ID XU2026

What this study shows

In 71 children with kaposiform haemangioendothelioma without Kasabach-Merritt phenomenon, head and neck lesions achieved a 100% 12-month radiologic response to sirolimus monotherapy versus 76% elsewhere; anatomical location was the only independent predictor of response. Observational: a matched multicentre cohort, not a randomised comparison, so confounding by indication and by lesion characteristics cannot be excluded and the association between site and response is not established as causal.

Abstract

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Sirolimus is the cornerstone therapy for Kaposiform hemangioendothelioma (KHE). This multicentre study evaluated whether anatomical location independently predicts radiologic response to sirolimus monotherapy in KHE without Kasabach-Merritt phenomenon (KMP). 21 patients with head and neck KHE without KMP and 50 matched patients with non-head and neck KHE were treated with sirolimus monotherapy across five tertiary referral centres in China.

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At a glance

Evidence type H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
Study type3 - Human Observational
Model systemHuman (paediatric multicentre matched cohort, n=71)
JournalPediatric Blood & Cancer
Year2026
Peer reviewedYes
Record last updated2026-08-22
SourceDOI 10.1002/1545-5017.70635

Extracted findings

InterventionSirolimus monotherapy
TargetmTOR
ModelHuman
Effect100% 12-month radiologic response and 81% near-complete involution in head/neck KHE; no progression

Cite this paper

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Xu, Y., et al. (2026). High Responsiveness of Head and Neck Kaposiform Hemangioendothelioma Without Kasabach-Merritt Phenomenon to Sirolimus Monotherapy. Pediatric Blood & Cancer. https://doi.org/10.1002/1545-5017.70635

@article{XU2026,
  author       = {Xu, Y. and Zhou, J. and Lan, Y. and others},
  title        = {{High Responsiveness of Head and Neck Kaposiform Hemangioendothelioma Without Kasabach-Merritt Phenomenon to Sirolimus Monotherapy}},
  journal      = {Pediatric Blood & Cancer},
  year         = {2026},
  doi          = {10.1002/1545-5017.70635},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper. 

Barton, O. (2026). Oliver's mTOR Atlas (record XU2026) [Data set]. https://mtor-atlas.org/study/XU2026/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_XU2026,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record XU2026},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/XU2026/},
  doi          = {10.5281/zenodo.22059963}
}