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Treatment practices and response in Kaposiform hemangioendothelioma: a multi-center cohort study
  • +21
  • Alexandra Borst,
  • Whitney Eng,
  • Morgan Griffin,
  • Kiersten Ricci,
  • Elissa Engel,
  • Denise Adams,
  • Jillian Dayneka,
  • Sally J. Cohen-Cutler,
  • Steven M. Andreoli,
  • Melinda D. Wu,
  • Allison P. Wheeler,
  • Kenneth Heym,
  • Shelley Crary,
  • Taizo Nakano,
  • Rachael Schulte,
  • Bhuvana Setty,
  • Thomas McLean,
  • Kristy S. Pahl,
  • Stefanos Intzes,
  • Irina Pateva,
  • Max Teitelbaum,
  • Zili Zong,
  • Yimei Li,
  • Michael Jeng
Alexandra Borst
The Children's Hospital of Philadelphia

Corresponding Author:[email protected]

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Whitney Eng
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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Morgan Griffin
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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Kiersten Ricci
Cincinnati Children's Hospital Medical Center
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Elissa Engel
Cincinnati Children's Hospital Medical Center
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Denise Adams
The Children's Hospital of Philadelphia
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Jillian Dayneka
The Children's Hospital of Philadelphia
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Sally J. Cohen-Cutler
Children's Hospital Los Angeles
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Steven M. Andreoli
Nemours Children's Specialty Care Jacksonville
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Melinda D. Wu
Oregon Health & Science University Doernbecher Children's Hospital
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Allison P. Wheeler
Monroe Carell Junior Children's Hospital at Vanderbilt
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Kenneth Heym
Cook Children's Medical Center
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Shelley Crary
Arkansas Children's Hospital
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Taizo Nakano
Children's Hospital Colorado
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Rachael Schulte
Riley Children's Foundation
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Bhuvana Setty
Nationwide Children's Hospital
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Thomas McLean
Atrium Health Wake Forest Baptist
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Kristy S. Pahl
Duke Children's Hospital and Health Center
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Stefanos Intzes
Providence Sacred Heart Medical Center
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Irina Pateva
University Hospitals Rainbow Babies & Children's Hospital
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Max Teitelbaum
Byram Hills High School
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Zili Zong
The Children's Hospital of Philadelphia
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Yimei Li
The Children's Hospital of Philadelphia
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Michael Jeng
Lucile Salter Packard Children's Hospital at Stanford General Pediatrics
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Background and Objectives: Kaposiform hemangioendothelioma (KHE) and tufted angioma (TA) are rare vascular tumors in children historically associated with significant morbidity and mortality. This study was conducted to determine first line therapy in the absence of available prospective clinical trials. Methods: Patients from 17 institutions diagnosed with KHE/TA between 2005-2020 with > 6 months follow-up were included. Response rates to sirolimus and vincristine were compared at 3 and 6 months. Durability of response and response to other treatment modalities were also evaluated. Results: Of 159 unique KHE/TA subjects, Kasabach-Merritt phenomenon (KMP) was present in 64 (40.3%) and only 2 patients were deceased (1.3%). Over 60% (n=96) demonstrated treatment response at 3 months and >70% (n=114) by 6 months (no significant difference across groups). The vincristine group had higher radiologic response at 3 months compared to sirolimus (72.7% vs 20%, p=0.03) but there was no differences between these groups at 6 months. There were no differences in rates of recurrent or progressive disease between vincristine and sirolimus. Conclusions: In this large, multicenter cohort of 159 patients with KHE/TA, rates of KMP were consistent with historical literature but the mortality rate (1.3%) was much lower. Overall treatment response rates were high (>70%) and there were no significant difference in treatment response or durability of disease comparing sirolimus to vincristine. Our results support individualized treatment decision plans depending on clinical scenario and patient/physician preferences. Response criteria and response rates reported here will be useful for guiding future treatment protocols for vascular tumors.
12 Aug 2023Submitted to Pediatric Blood & Cancer
12 Aug 2023Submission Checks Completed
12 Aug 2023Assigned to Editor
12 Aug 2023Review(s) Completed, Editorial Evaluation Pending
16 Aug 2023Reviewer(s) Assigned
06 Oct 2023Editorial Decision: Revise Major
25 Oct 20231st Revision Received
25 Oct 2023Submission Checks Completed
25 Oct 2023Assigned to Editor
25 Oct 2023Review(s) Completed, Editorial Evaluation Pending
26 Oct 2023Reviewer(s) Assigned
08 Nov 2023Editorial Decision: Accept