Stem Cell Transplantation Committee (SCT)

Committee Chairman:

Adriana Balduzzi

Committee Members:

Persis Amrolia, Marc Ansari, Florian Babor, Peter Bader, Adriana Balduzzi, Alice Bertaina, Ettore Biagi, Bella Bielorai, Marc Bierings, Jochen Büchner, Birgit Burkhardt, Selim Corbacioglu, Jean-Hugues Dalle, Giovanna D’Amico, Erica Dander, Brenda Gibson, Sara Ghorashian, Gal Goldstein, Sergio Gomez, Stelios Graphakos, Rupert Handgretinger, Carsten Heilmann, Ingeborg Hirsh, Peter Hoogerbrugge, Marianne Ifversen, Koji Kato, Nagy Kalman, Thomas Klingebiel, Edoardo Lanino, Arjan Lankester, Anita Lawitschka, Vincent Lee, Franco Locatelli, Giovanna Lucchini, Chiara Magnani, Susanne Matthes-Martin, Roland Meisel, Karin Mellgren, Gérard Michel, Giuseppe Morreale, Ingo Müller, Julia Palma, Christina Peters, Arnaud Petit,   Anna Pieczonka, Michael Pulsipher, Claudia Rossig, Attilio Rovelli, Martin Sauer, Peter Shaw, Kirk Schultz, Petr Sedlacek, Jan Stary, Jerry Stein, Polina Stepensky, Jacek Toporski, Amos Toren, Dominik Turkiewicz, Kim Vettenranta, Jacek Wachowiak, Andre Manfred Willasch, Isaac Yaniv

Clinical Trials:

Open:

ALL SCT BFM International, Allogeneic Stem Cell Transplantation for Children and Adolescents with ALL, Prospective international controlled comparative non-randomized study:

  • Principal investigator: Christina Peters, Email: Christina.peters@stanna.at.
  • Start: January 2007.
  • Participating countries: Austria, Chile, Czech Republic, Denmark, France, Israel, Italy, The Netherlands, Poland, Slowakia, Sweden, Turkey.
  • The study is closed for patient registration; follow up and data analyses are ongoing

ALL SCT PED 2012 Forum (for omitting radiation under majority age). Allogeneic Stem Cell Transplantation in Children and Adolescents with ALL in CR. Prospective controlled randomized international study:

  • Principal investigator: Christina Peters, E-mail: christina.peters@stanna.at
  • Participating countries: Austria, Argentina, Australia, Chile, Czech Republic. Denmark, Finland, France, Germany, Greece, Hong-Kong, Hungary, Japan, Israel, Italy, The Netherlands, New Zealand, Norway, Polland, Slovakia, Sweden, Spain, Switzerland, Turkey, United Kingdom, USA
  • The study is open for international recruitment. 398 patients planned; issue: randomization rate

CML SCT I-BFM: Multicenter Study: Prospective controlled multi-center therapy optimization study for children with CML undergoing SCT. Prospective international single-arm study.

  • Main investigator: Susanne Matthes-Martin, Email: susanne.matthes@stanna.at
  • Active countries: Austria, Czech Republic, Germany, Israel, Italy, Poland. Accrual: 13 enrolled patients, + 12 observational patients. Outcome on 25 patients: alive 24/25, deaths in CR 1/25, TKI-free alive 22/25. Closure planned in 2017.
  • The study is open for international recruitment since 2009.

Role of MRD in ALL after SCT: Retrospective observational multi-center WIG joint I-BFM/EBMT and COG/PBMTC study for assessing the role of post-transplant MRD in children with ALL:

  • Main investigators: Peter Bader & Michael Pulsipher, Email: peter.bader@kgu.de, mpulsipher@chla.usc.edu.
  • Active countries: Australia, Austria, Germany, Italy, The Netherlands, Switzerland, United States of America. Accrual: 748 patients.
  • Status: Analyses ongoing.

Refractory NHL management within I-BFM. A retrospective I-BFM study:

  • Main investigators: Birgit Burkhardt, Email: birgit.burkhardt@ukmuenster.de
  • Active countries: Australia, Austria, Belarus, Czech Republic, France, Finland, Germany,  Hong-Kong, Italy, The Netherlands, Poland, Russia, Slovakia, Switzerland, Turkey, United Kingdom, United States of America. Accrual: 514 patients
  • Status: Analyses ongoing.

AML post transplant relapse. A retrospective I-BFM study:

  • Main investigators: Martin Sauer, Email: sauer.martin@mh-hannover.de
  • Active countries: Australia, Denmark, Italy, Netherlands, Belgium, Israel, Sweden, Norway, Finland, Austria, Czech Republic, Germany. Accrual: 289 patients (175 non-transplanted, 105 transplanted).
  • Analyses ongoing.

AML eligibility criteria for transplantation: an I-BFM Survey. Survey circulated among SCT Committee Members regarding SCT eligibility criteria in AML pediatric patients:

  • Main investigator: Brenda Gibson, Email: brenda.Gibson@ggc.scot.nhs.uk
  • Participating groups: AIEOP, BFM, Canada, COG, CPH, DCOG, France, Gazi, Israel, JCCG / JPLSG, NOPHO, PINDA, PLLSG, Serbia, Slovenia, St.Jude, UK. Accrual: 18 countries
  • Status: Manuscript in preparation

Completed:

N/A

Activities/Projects:

In the prospective study ALL SCT BFM International, the committee enrolled 410 patients to prospectively evaluate whether allogeneic SCT from a matched donor is equivalent to allogeneic SCT from an HLA-identical sibling, as well as evaluate the transplantation from mismatched donors (8/10, unrelated cord blood, haploidentical). Multiple publications are now in preparation (core comparison MSD versus MD; comparison MMD versus MD; MMD per se; impact of graft cell counts;GVHD).

The  study ALL SCT PED 2012 Forum (for omitting radiation under majority age) enrolled 384 patients so far, 198 of whom have been randomized (status as of January 2017). Its objectives are:

  • To show that a non TBI containing conditioning (Flu/Thio/ivBu OR Flu/Thio/Treo) results in a non inferior survival as compared to conditioning with TBI/Etoposide in children older than 4 years after HSCT from an HLA identical sibling donor (MSD) or a HLA matched unrelated donor (MUD).
  • To explore EFS after HSCT from HLA mismatched donors MMD) using mmUD, mmCB or HLA haploidentical family members with non TBI-conditioning regimen
  • To explore whether particular sub-groups of patients have lower randomisation-rates and whether outcome (EFS and Overall-Survival) in randomised and non-randomised patients is different.
  • To explore pre-defined risk-factors and treatment by risk-factor interactions of prospectively defined adverse events of special interest (AESIs) in more detail

The prospective international single-arm study CML SCT I-BFM on multi-center therapy optimization study for children with CML undergoing SCT aimed to:

  • Evaluate 1-year transplant-related mortality of children and adolescents with CML and matched related or unrelated donors following reduced-intensity conditioning.
  • Evaluate 5-year event-free and current-leukemia-free survival without TKI-therapy in patients undergoing frequent molecular disease monitoring with early immunological intervention.

The retrospective observational multi-center WIG joint I-BFM/EBMT and COG/PBMTC study for assessing the role of post-transplant MRD in children with ALL aimed to:

  • Assess the impact of post-transplant MRD on outcome in an international setting;
  • Evaluate the association between GVHD and relapse in MRD positive patients after transplant

The retrospective I-BFM study on refractory NHL management aimed to assess strategies adopted in relapsed/refractory non Hodgkin lymphoma.

The retrospective AML post transplant relapse study aims to assess the strategies adopted in post-transplant relapse in AML an to create a working network as a platform for prospective  studies.

Finally, the AML eligibility criteria for transplantation survey aims to define the SCT eligibility criteria in AML paediatric patients for the purpose of investigation/harmonization.

Recent Publications:

N/A

Meetings:

Recent:

  • SCT Committee Sessions, Jerusalem, Israel, 10-12 May 2017
  • SCT Committee I-BFM Interim Meeting, Milan, Italy, January 23-24, 2018

Next:

N/A

Contact:

Adriana Balduzzi    abalduzzi@fondazionembbm.it