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Children
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Advances in the Epidemiology of Hemostasis Disorders in Children
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Advances in the Epidemiology of Hemostasis Disorders in Children

A special issue of Children (ISSN 2227-9067). This special issue belongs to the section "Pediatric Hematology & Oncology".

Deadline for manuscript submissions: 30 May 2026 | Viewed by 817

Special Issue Editors

Prof. Dr. Anca Colita

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Guest Editor
Department of Pediatrics, Faculty of Medicine, “Carol Davila” University of Medicine and Pharmacy, 050474 Bucharest, Romania
Interests: pediatric hematology; bone marrow transplantation; bleeding disorders; coagulation disorders; general pediatrics
Special Issues, Collections and Topics in MDPI journals
Prof. Dr. Cristina Gavrilovici

E-Mail Website
Guest Editor
Pediatric Department, University of Medicine and Pharmacy "Grigore T. Popa", 16 University Street, 700115 Iasi, Romania
Interests: pediatric hematology; bleeding disorders; coagulation disorders; general pediatrics
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Hemostatic disorders in children, whether congenital or acquired, encompass a broad and complex spectrum of conditions associated with substantial morbidity and mortality. Beyond classical bleeding disorders—such as hemophilia, von Willebrand disease, platelet function abnormalities and vascular or connective tissue syndromes—disturbances of hemostasis may also manifest as thrombotic or microangiopathic complications, including cerebral or venous thromboses, disseminated intravascular coagulation (DIC), thrombotic thrombocytopenic purpura (TTP) and hemolytic uremic syndrome (HUS).

The epidemiology and underlying mechanisms of these disorders remain heterogeneous and incompletely elucidated. Recent advances in molecular genetics, biomarker identification and precision diagnostics have profoundly expanded our understanding of the pathways driving both bleeding and thrombosis in pediatric populations.

A comprehensive characterization of hemostatic disorders—from neonatal hemostasis to long-term complications of chronic diseases—requires an integrated, multidisciplinary approach involving hematologists, pediatricians, geneticists, immunologists and basic and translational researchers.

In this Special Issue, we welcome original research articles, reviews and case reports addressing advances in the epidemiology, diagnosis and management of pediatric hemostatic disorders. Contributions exploring molecular mechanisms, innovative therapeutic approaches and preventive strategies are particularly encouraged.

We look forward to your valuable contributions.

Prof. Dr. Anca Colita
Prof. Dr. Cristina Gavrilovici
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Children is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2400 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • pediatric hematology
  • hemostatic disorders
  • bleeding disorders
  • coagulation abnormalities
  • pediatric thrombosis
  • thrombotic microangiopathies
  • hemolytic uremic syndrome (HUS)
  • thrombotic thrombocytopenic purpura (TTP)
  • molecular and genetic mechanisms
  • precision diagnostics

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Published Papers (1 paper)

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Research

22 pages, 979 KB  
Article
Case Series and Literature Narrative Review of Immune-Mediated Thrombotic Thrombocytopenic Purpura in Children
by Letiția-Elena Radu, Andreea Nicoleta Șerbănică, Andra Daniela Marcu, Ana-Maria Bică, Cristina Georgiana Jercan, Radu Obrișcă, Georgiana Gherghe, Gabriela Droc, Dana Tomescu and Anca Coliță
Children 2026, 13(3), 350; https://doi.org/10.3390/children13030350 - 28 Feb 2026
Viewed by 587
Abstract
Background/Objectives: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare but life-threatening thrombotic microangiopathy in children. Secondary forms, occurring in association with immune dysregulation, autoimmune disease, or other triggers, are particularly challenging to diagnose and manage, and pediatric-specific data remain limited. This study [...] Read more.
Background/Objectives: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare but life-threatening thrombotic microangiopathy in children. Secondary forms, occurring in association with immune dysregulation, autoimmune disease, or other triggers, are particularly challenging to diagnose and manage, and pediatric-specific data remain limited. This study aimed to describe the clinical characteristics, diagnostic pathways, and management of pediatric iTTP and to contextualize these findings within the recent literature. Methods: We conducted a retrospective case series of pediatric patients diagnosed with iTTP at a tertiary referral center, between November 2021 and January 2026. Clinical presentation, laboratory findings, including ADAMTS13 activity and ADAMTS13 inhibitors, associated conditions, treatment strategies, and outcomes were reviewed. In parallel, a narrative literature review was performed focusing on pediatric immune-mediated secondary TTP published over the past five years. Results: Four pediatric patients (three females, one male; median age 14 years) met inclusion criteria. All presented with severe thrombocytopenia and microangiopathic hemolytic anemia, accompanied by prominent neurologic manifestations in three cases. Severe ADAMTS13 activity deficiency (≤10%) with positive inhibitors was documented in all patients. Secondary iTTP occurred in association with evolving systemic autoimmunity, systemic lupus erythematosus, common variable immunodeficiency, or without an identifiable trigger at presentation. High clinical probability scores facilitated early diagnosis. Management required plasma exchange, corticosteroids, and targeted and immunomodulatory therapy. Conclusions: Pediatric secondary iTTP is a heterogeneous condition that frequently presents with diagnostic ambiguity and severe neurologic involvement. Early recognition, prompt initiation of TTP-directed therapy, and comprehensive immunologic evaluation are critical for favorable outcomes. Case series combined with narrative reviews remain valuable for advancing understanding and optimizing individualized care in this rare pediatric disorder. Full article
(This article belongs to the Special Issue Advances in the Epidemiology of Hemostasis Disorders in Children)
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