A pilot study combining primary busulfan-based haploidentical stem cell transplantation with GD2 antibody to treat high-risk neuroblastoma - Scorecard - MDSpire

A pilot study combining primary busulfan-based haploidentical stem cell transplantation with GD2 antibody to treat high-risk neuroblastoma

  • By

  • Sveva Castelli

  • Tim Flaadt

  • Franziska Schulze

  • Theresa M. Thole-Kliesch

  • Felix Zirngibl

  • Lena Oevermann

  • Annette Künkele

  • Kathy Astrahantseff

  • Stefanie Schulte

  • Louisa Duell

  • Joerg Fuchs

  • Thorsten Simon

  • Barbara Hero

  • Patrick Hundsdoerfer

  • Julian M. M. Rogasch

  • Peter Lang

  • Steven Warmann

  • Arend von Stackelberg

  • Johannes H. Schulte

  • Angelika Eggert

  • Hedwig E. Deubzer

  • May 29, 2026

  • 0 min

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Clinical Scorecard: A preliminary investigation of haploidentical stem cell transplantation using a busulfan regimen combined with GD2 antibody therapy for the management of high-risk neuroblastoma

At a Glance

CategoryDetail
Condition
Key MechanismsBusulfan/melphalan conditioning, haploidentical stem cell transplantation, GD2-directed antibody therapy
Target Population
Care Setting

Key Highlights

  • Event-free survival is <20% in very high-risk neuroblastoma patients.
  • Haploidentical stem cell transplantation (haplo-SCT) shows feasibility and effectiveness.
  • Three of five patients remained in complete remission for over 1.5 years post-transplant.
  • Further confirmation in larger controlled trials is warranted.

Guideline-Based Recommendations

Diagnosis

    Management

    • Use of haplo-SCT combined with dinutuximab beta for high-risk neuroblastoma.

    Monitoring & Follow-up

      Risks

        Patient & Prescribing Data

        Five pediatric patients with INRG stage M high-risk neuroblastoma.

        Patients received conditioning with busulfan/melphalan followed by haplo-SCT and GD2 antibody therapy.

        Clinical Best Practices

        • Ensure informed consent is obtained from parents or guardians.
        • Conduct data collection in accordance with ethical standards.

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        Original Source(s)

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