Next Article in Journal
Markers of Antiseizure Treatment Efficacy in Children: A Review
Previous Article in Journal
Association Between Surgical Timing and Perioperative Outcomes in Lumbar Spine Surgery: A Retrospective Cohort Study
 
 
Search for Articles:
Title / Keyword
Author / Affiliation / Email
Journal
Article Type
 
 
Advanced Search
 
Section
Special Issue
Volume
Issue
Number
Page
 
Journals
JCM
Volume 15
Issue 11
10.3390/jcm15114190
jcm-logo
Submit to this Journal Review for this Journal Propose a Special Issue
► Article Menu

Article Menu

share Share announcement Help format_quote Cite question_answer Discuss in SciProfiles
first_page
settings
Order Article Reprints
Font Type:
Arial Georgia Verdana
Font Size:
Aa Aa Aa
Line Spacing:
Column Width:
Background:
This is an early access version, the complete PDF, HTML, and XML versions will be available soon.
Case Report

Begelomab (BEGESAND®) Salvages Steroid-Resistant Acute GVHD in Pediatric Patients

by
David Shyr
1,*,
Steve Chirielesion
1,
Sebastian Fernandez-Pol
1,
Katja Weinacht
1,
Rajni Agarwal
1,
Ami J. Shah
1,
Michela Spinelli
2,
Renata Palmieri
2,
Antonio Francesco Di Naro
2 and
Alice Bertaina
1
1
School of Medicine, Stanford University, Stanford, CA 94305, USA
2
Adienne Pharma & Biotech, 6900 Lugano, Switzerland
*
Author to whom correspondence should be addressed.
J. Clin. Med. 2026, 15(11), 4190; https://doi.org/10.3390/jcm15114190
Submission received: 19 April 2026 / Revised: 20 May 2026 / Accepted: 22 May 2026 / Published: 28 May 2026
(This article belongs to the Special Issue New Developments in Hematopoietic Stem Cell Transplantation in Clinical Practice)
Versions Notes

Abstract

Background: Acute graft-versus-host disease (aGVHD) is a leading cause of morbidity and mortality following pediatric hematopoietic stem cell transplantation (HSCT). Approximately half of children achieve complete response (CR) to corticosteroids, whereas steroid-refractory (SR) disease carries a 1–2-year mortality of 41–44%. Mortality risk is 2.6-fold higher in children >13.9 years, and respiratory failure accounts for 26% of deaths. Existing second-line agents—ruxolitinib, tocilizumab, or extracorporeal photopheresis—have delayed onset or high toxicity. Begelomab (BEGESAND®), a monoclonal antibody targeting CD26/dipeptidyl peptidase-4 (DPP4), inhibits CD26-mediated T-cell activation and has demonstrated 75% response in adults with minimal toxicity. However, pediatric data are lacking. Methods: We retrospectively reviewed five consecutive pediatric patients (ages 3–20 years) treated with Begelomab (BEGESAND®) for SR (n = 4) or steroid-dependent (SD; n = 1) aGVHD between 2017–2021 under emergency IND authorization. Begelomab (BEGESAND®) was administered intravenously at 2.7 mg/m2/day on days 1–5, 10, 14, 17, 21, 24, and 28. GVHD was graded by MAGIC criteria; flow cytometry and immunohistochemistry (IHC) assessed CD26 expression and immune effects. Results: All patients had grade IV disease after ≥2 prior agents. Two with pre-existing sepsis died early, before treatment response could be assessed. Of three evaluable patients, two (67%) achieved CR within 21 days and one achieved durable control by 6 months. All three remain alive; no Begelomab (BEGESAND®)-related toxicity, cytopenia, or new infections occurred. Flow cytometry showed preserved T-cell subsets, and IHC demonstrated CD26 localization at sites of epithelial injury. Conclusions: Begelomab (BEGESAND®) showed promising timely and durable responses with excellent safety in pediatric SR/SD-aGVHD, supporting further evaluation in multicenter pediatric trials.
Keywords: Begelomab; graft-versus-host disease; stem cell transplant Begelomab; graft-versus-host disease; stem cell transplant

Share and Cite

MDPI and ACS Style

Shyr, D.; Chirielesion, S.; Fernandez-Pol, S.; Weinacht, K.; Agarwal, R.; Shah, A.J.; Spinelli, M.; Palmieri, R.; Di Naro, A.F.; Bertaina, A. Begelomab (BEGESAND®) Salvages Steroid-Resistant Acute GVHD in Pediatric Patients. J. Clin. Med. 2026, 15, 4190. https://doi.org/10.3390/jcm15114190

AMA Style

Shyr D, Chirielesion S, Fernandez-Pol S, Weinacht K, Agarwal R, Shah AJ, Spinelli M, Palmieri R, Di Naro AF, Bertaina A. Begelomab (BEGESAND®) Salvages Steroid-Resistant Acute GVHD in Pediatric Patients. Journal of Clinical Medicine. 2026; 15(11):4190. https://doi.org/10.3390/jcm15114190

Chicago/Turabian Style

Shyr, David, Steve Chirielesion, Sebastian Fernandez-Pol, Katja Weinacht, Rajni Agarwal, Ami J. Shah, Michela Spinelli, Renata Palmieri, Antonio Francesco Di Naro, and Alice Bertaina. 2026. "Begelomab (BEGESAND®) Salvages Steroid-Resistant Acute GVHD in Pediatric Patients" Journal of Clinical Medicine 15, no. 11: 4190. https://doi.org/10.3390/jcm15114190

APA Style

Shyr, D., Chirielesion, S., Fernandez-Pol, S., Weinacht, K., Agarwal, R., Shah, A. J., Spinelli, M., Palmieri, R., Di Naro, A. F., & Bertaina, A. (2026). Begelomab (BEGESAND®) Salvages Steroid-Resistant Acute GVHD in Pediatric Patients. Journal of Clinical Medicine, 15(11), 4190. https://doi.org/10.3390/jcm15114190

Note that from the first issue of 2016, this journal uses article numbers instead of page numbers. See further details here.

Article Metrics

Back to TopTop