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

Xu Y, Zhou J, Lan Y, Yang L, Zhang Y, Ji Y · 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

ShowHide

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.

Read the full abstract on PubMed →

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 type	3 - Human Observational
Model system	Human (paediatric multicentre matched cohort, n=71)
Journal	Pediatric Blood & Cancer
Year	2026
Peer reviewed	Yes
Record last updated	2026-10-01
Source	DOI 10.1002/1545-5017.70635 · PMID 42657977

Extracted findings

These fields were extracted from the paper with AI assistance and have not been checked against the source. Read them as a pointer into the paper, not as curated claims; where they disagree with the curated summary above, the curated summary stands.

Intervention	Sirolimus monotherapy
Target	mTOR
Model	Human
Effect	100% 12-month radiologic response and 81% near-complete involution in head/neck KHE; no progression

In the Atlas

Cite this paper

ShowHide

Xu, Y., Zhou, J., Lan, Y., Yang, L., Zhang, Y., & Ji, Y. (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 Yang, L. and Zhang, Y. and Ji, Y.},
  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},
  note         = {PMID: 42657977},
}

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}
}

Was this page useful?