Clinical trial NCT06927440: National Multicenter Registry for Pediatric End-Stage Heart Failure in China
Official title: Development and Implementation of a National Multicenter Registry for Pediatric End-Stage Heart Failure in China
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Heart Failure
Children
Keywords: end-stage heart failure
pediatric
Lead sponsor: Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
Collaborators: name: Ministry of Science and Technology of the People´s Republic of China
First posted: 2025-04-15
Enrollment: 1000
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with end-stage heart failure (ESHF) receiving care at multiple Chinese tertiary referral centers

Brief summary: To establish a multicenter pediatric end-stage heart failure (ESHF) specialized disease data platform and promote its adoption across major children's medical centers nationwide. Based on this platform, conduct a national multicenter cohort study on specialized treatments for pediatric ESHF (including pharmacotherapy, ventricular assist devices, heart transplantation, etc.). This initiative will provide a foundational platform for advancing national clinical research on pediatric end-stage heart failure.


Eligibility criteria: Inclusion Criteria:

Patients who meet any of the following criteria despite receiving optimal guideline-directed medical therapy (GDMT):

Severe and persistent heart failure symptoms \[New York Heart Association (NYHA) Class III (advanced) or IV\];

Significant cardiac dysfunction, defined as:

Left ventricular ejection fraction ≤30%

Isolated right ventricular failure

Non-operable severe valvular or congenital abnormalities

Persistently elevated B-type natriuretic peptide (BNP) or N-terminal pro-BNP (NT-proBNP) levels

Severe diastolic dysfunction or left ventricular structural abnormalities (based on ESC definitions for heart failure with preserved ejection fraction \[HFpEF\] and heart failure with mildly reduced ejection fraction \[HFmrEF\]);

Requirement of:

High-dose intravenous diuretics (or combination diuretic therapy) for pulmonary/systemic congestion, OR

Inotropic/vasoactive agents for low cardiac output, OR

Management of malignant arrhythmias Resulting in \>1 unplanned hospital visits or admissions within the past 12 months;

Severely impaired mobility due to cardiac causes with either:

Inability to ambulate, OR

6-minute walk distance \<300 m, OR

Peak oxygen consumption (PvO₂) \<12-14 mL/kg/min. -

Exclusion Criteria:

Concurrent life-limiting systemic disorders

Structural cardiac lesions with indication for surgical/interventional correction -

Primary outcomes:
measure: Composite of cardiovascular death, heart transplantation, LVAD implantation or WHF hospitalization
timeFrame: From enrollment to the follow up at 1 year, 3 years, 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai Children's Medical Center
state: Shanghai
Study officials:
References:
citation: McDonagh TA, Metra M, Adamo M, Gardner RS, Baumbach A, Bohm M, Burri H, Butler J, Celutkiene J, Chioncel O, Cleland JGF, Crespo-Leiro MG, Farmakis D, Gilard M, Heymans S, Hoes AW, Jaarsma T, Jankowska EA, Lainscak M, Lam CSP, Lyon AR, McMurray JJV, Mebazaa A, Mindham R, Muneretto C, Francesco Piepoli M, Price S, Rosano GMC, Ruschitzka F, Skibelund AK; ESC Scientific Document Group. 2023 Focused Update of the 2021 ESC Guidelines for the diagnosis and treatment of acute and chronic heart failure. Eur Heart J. 2023 Oct 1;44(37):3627-3639. doi: 10.1093/eurheartj/ehad195. No abstract available.
citation: Heidenreich PA, Bozkurt B, Aguilar D, Allen LA, Byun JJ, Colvin MM, Deswal A, Drazner MH, Dunlay SM, Evers LR, Fang JC, Fedson SE, Fonarow GC, Hayek SS, Hernandez AF, Khazanie P, Kittleson MM, Lee CS, Link MS, Milano CA, Nnacheta LC, Sandhu AT, Stevenson LW, Vardeny O, Vest AR, Yancy CW; ACC/AHA Joint Committee Members. 2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure: A Report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. Circulation. 2022 May 3;145(18):e895-e1032. doi: 10.1161/CIR.0000000000001063. Epub 2022 Apr 1.