A pilot study combining primary busulfan-based haploidentical stem cell transplantation with GD2 antibody to treat high-risk neuroblastoma - Report - 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 Report: Haploidentical Stem Cell Transplantation for High-Risk Neuroblastoma

Overview

This preliminary investigation evaluates haploidentical stem cell transplantation (haplo-SCT) combined with a busulfan regimen and GD2 antibody therapy in pediatric patients with high-risk neuroblastoma. Early results indicate feasibility and potential survival benefits, with three out of five patients remaining in complete remission for extended periods.

Background

Very high-risk neuroblastoma presents significant treatment challenges, with event-free survival rates below 20%. Traditional therapies often fail to mobilize sufficient hematopoietic stem cells for effective treatment. Haploidentical stem cell transplantation offers a promising alternative, leveraging graft-versus-tumor effects and immunotherapy to improve outcomes in this vulnerable patient population.

Data Highlights

Patient OutcomesDetails
Patients Treated5
Patients Alive in Remission3
Years in Remission7.3, 6.3, 1.5
Events1 relapse, 1 non-relapse death

Key Findings

  • Haplo-SCT combined with busulfan/melphalan and GD2-directed immunotherapy is feasible.
  • All patients in the pilot cohort successfully engrafted.
  • Three out of five patients achieved first complete remission lasting 1.5 to 7.3 years.
  • Two patients experienced adverse events, including one relapse and one non-relapse death.
  • Early results suggest a potential survival benefit for very high-risk pediatric neuroblastoma patients.

Clinical Implications

The findings support the exploration of haploidentical stem cell transplantation as a viable treatment option for pediatric patients with high-risk neuroblastoma. Further investigation in larger controlled trials is necessary to confirm these preliminary results and assess long-term outcomes.

Conclusion

This pilot study indicates that haploidentical stem cell transplantation combined with a busulfan regimen and GD2 antibody therapy may offer a promising approach for managing high-risk neuroblastoma in children. Larger trials are needed to validate these findings.

Related Resources & Content

  1. Blood Cancer Journal, 2024 -- Busulfan-fludarabine versus busulfan-cyclophosphamide for allogeneic transplant in acute myeloid leukemia: long term analysis of GITMO AML-R2 trial
  2. Bone Marrow Transplantation, 2024 -- Efficacy of a novel myeloablative treatment combining gemcitabine, clofarabine, and busulfan in allogeneic transplantation for aggressive lymphomas
  3. Bone Marrow Transplantation, 2024 -- Haploidentical Hematopoietic Stem Cell Transplantation in Hematologic Cancers: A New Conditioning Approach Utilizing Anti-T Lymphocyte Immunoglobulin for In Vivo T Cell Depletion
  4. Neuroblastoma Treatment (PDQ®) - NCI, 2023 -- Neuroblastoma Treatment
  5. Long-term follow-up of a Phase III Study of ch14.18 (Dinutuximab) + Cytokine Immunotherapy in Children with High-risk Neuroblastoma: Children’s Oncology Group Study ANBL0032
  6. Bone Marrow Transplantation — Rescue HLA-haploidentical hematopoietic stem cell transplantation utilizing post-transplant cyclophosphamide for addressing graft failure in non-malignant conditions
  7. Neuroblastoma Treatment (PDQ®) - NCI
  8. Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations
  9. Neuroblastoma Treatment (PDQ®) - NCI
  10. Long-term follow-up of a Phase III Study of ch14.18 (Dinutuximab) + Cytokine Immunotherapy in Children with High-risk Neuroblastoma: Children’s Oncology Group Study ANBL0032 - PMC
  11. Efficacy and Safety of Anti-GD2 Immunotherapy with Dinutuximab Beta in the Treatment of Relapsed/Refractory High-Risk Neuroblastoma - PMC
  12. Fcγ Receptor Polymorphism in Patients with Relapsed/Refractory High-Risk Neuroblastoma Correlates with Outcomes in the SIOPEN Dinutuximab Beta Long-Term Infusion Trial - PubMed
  13. Telomere maintenance mechanisms in neuroblastoma: New insights and translational implications - ScienceDirect
  14. Anti-GD2 Antibody Dinutuximab Beta and Low-Dose Interleukin 2 After Haploidentical Stem-Cell Transplantation in Patients With Relapsed Neuroblastoma: A Multicenter, Phase I/II Trial - PMC
  15. Haploidentical stem cell transplantation in newly diagnosed high-risk neuroblastoma: Results from a single-arm, prospective study from India - PubMed
  16. Haploidentical Stem Cell Transplantation Using PTCY Platform in Paediatric Patients with High-Risk Neuroblastoma - ScienceDirect

Original Source(s)

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

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