Early complications following haematopoietic SCT in children

M Miano1, M Faraci, G Dini

  • 1Department Haematology/Oncology, G Gaslini Children's Research Institute, Genoa, Italy. mauriziomiano@ospedale-gaslini.ge.it

Insights

Early transplant complications like VOD, TAM, engraftment syndrome, and HC stem from cellular damage. Defibrotide shows promise for VOD, while other conditions require supportive care or specific treatments.

Area of Science:

  • Hematology
  • Transplantation Medicine
  • Pediatric Oncology

Background:

  • Early complications after hematopoietic stem cell transplant (HSCT) occur within 100 days.
  • Epithelial and endothelial damage are the primary causes of these frequent complications.
  • Clinical manifestations vary based on the affected area and severity of endothelial damage.

Purpose of the Study:

  • To review the pathogenesis, clinical features, and treatment of early HSCT complications.
  • To highlight the incidence and outcomes of specific early complications.
  • To discuss emerging therapeutic strategies and their current evidence.

Main Methods:

  • Literature review focusing on early complications post-HSCT.
  • Analysis of pathogenetic mechanisms, including endothelial and epithelial injury.
  • Summary of clinical presentations, diagnostic criteria, and treatment modalities.

Main Results:

  • Veno-occlusive disease (VOD) affects 27-40% of pediatric HSCT recipients, with defibrotide showing effectiveness but high mortality.
  • Thrombotic microangiopathy (TAM) occurs in 4-13%, often linked to immunosuppressants, requiring plasmapheresis.
  • Engraftment syndrome and hemorrhagic cystitis (HC) present with distinct symptoms and require specific management, with investigational therapies for viral HC.

Conclusions:

  • Early recognition and management of HSCT complications are crucial for patient outcomes.
  • Defibrotide is a key treatment for VOD, though further research is needed for TAM.
  • Multifaceted approaches, including supportive care and targeted therapies, are essential for managing diverse early post-transplant complications.

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