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New drug combo may make stem cell transplants safer for kids with blood disorders

NCT ID NCT02349906

First seen Jan 04, 2026 · Last updated May 01, 2026 · Updated 17 times

Summary

This study tested two different drug combinations given before a stem cell transplant in 106 children with non-cancerous diseases like immune deficiencies and blood disorders. The goal was to see if one drug (treosulfan) caused fewer serious side effects or deaths than the standard drug (busulfan). The approach uses chemotherapy to prepare the body for donor stem cells, but children may still need lifelong monitoring or medications afterward.

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Contacts and locations

Locations

  • Department of Pediatric Oncology & Hematology, Charite Berlin

    Berlin, 13353, Germany

  • Dzieciecy Szpital Kliniczny im. A. Gebali w Lublinie

    Lublin, 20-093, Poland

  • Hannover Medical University, Dep. of Paediatrics, Paediatric Haematology and Oncology

    Hanover, Germany

  • Heidelberg University Hospital

    Heidelberg, 69120, Germany

  • Oncoematologia Pediatrica A.O. di Perugia Ospedale S. Maria della Misericordia

    Perugia, 06156, Italy

  • Ospedale Bambino Gesu Roma

    Rome, 00165, Italy

  • Ospedale Infantile Regina Margherita Torino

    Turin, 10126, Italy

  • Reparto Trapianti Midollo Osseo Clinica Pediatrica Universitaria Fondazione MBBM-Ospedale San Gerardo

    Monza, 20900, Italy

  • S.C. Oncoematologia Pediatrica Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100, Italy

  • SC Oncoematologia Pediatrica Ospedale Pediatrico Microcitemico "Antonio Cao" A.O. Brotzu

    Cagliari, 09121, Italy

  • Szpital Uniwersytecki im. dr Antoniego Jurasza

    Bydgoszcz, 85-094, Poland

  • U.O. Oncoematologia Pediatrica Azienda Ospedaliero Universitaria Pisana Ospedale S. Chiara

    Pisa, 56100, Italy

  • U.O.C. Oncoematologia Pediatrica Policlinico "G.B. Rossi" - AOUI Verona

    Verona, 37134, Italy

  • UOC Ematologia ed Oncologia Pediatrica con TMO AOU Policlinico Vittorio Emanuele

    Catania, 95123, Italy

  • Ulm, University Hospital, Clinic for Children and Adolescents

    Ulm, 89075, Germany

  • University Children's Hospital Essen Pediatric stem cell transplantation

    Essen, 45122, Germany

  • University Hospital Frankfurt

    Frankfurt am Main, 60590, Germany

  • University Hospital Motol, Dep. of Paediatric Haematology and Oncology

    Prague, Czechia

  • University of Jena, Department of Pediatrics

    Jena, 07747, Germany

  • Uniwersytecki Szpital Dzieciecy w Krakowie

    Krakow, 30-663, Poland

  • Wroclaw Medical University, Department of Pediatric Hematology/Oncology and BMT

    Wroclaw, 50-368, Poland

Conditions

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