автор: Julia Dobke, erstellt am: 2014/06/19, Последнее изменение: 2015/06/11

титул Allogeneic Stem Cell Transplantation in Children and Adolescents with Acute Lymphoblastic Leukaemia
Формы рака Acute lymphblastic leukeamia
Вид исследования Open, randomised, multicentre, controlled, prospective phase III study
Цель исследования


Children an adolescents with lymphoblastic leukeamia can be cured with modern therapies in more than 80%. Nevertheless patients with high risk or relapsed acute lymphoblastic leukaemia (ALL) have a poor prognosis. For these patients intensive therapy is required after they have achieved remission with multimodal chemotherapy. Allogeneic hematopoietic stem cell transplantation (HSCT) can effectively induce immunological antileukaemic control in patients with ALL by means of the graft-versus-leukaemia effect (GvL), but treatment related mortality (TRM), morbidity and late effects remain serious problems of this treatment modality. In the last decade the short term outcome of 900 children with ALL from more than 60 different study centres who received allogeneic HSCT has improved, due to the use of donors more closely matched by Human leukocyte antigen (HLA) typing, resulting in less severe graft vs host disease (GvHD) and better supportive care. However, the risk of life long complications persists in all children. The observation and documentation of late complications are especially important for children and adolescents.


The biggest burdens for children given TBI are the risks of long term complications as secondary malignancies, growth retardation (especially if irradiated below 10 years) and infertility (most common after irradiation during or after puberty). This is why "ALL-SCTped Forum" explores the question, if TBI can be replaced by a less toxic combination of different chemotherapies.


Stratum 1

All patients > 4 years with a Matched Donor (MD) or Matched Sibling Donor (MSD) will be randomized between the conditioning with VP16/TBI or a Chemo-Conditioning (Busulfan/Thiatepa/Fludarabin or Treosulfan/Fludarabin/R´Thiotepa).

Stratum 2

Patients ≤ 4 years are not randomized and receive a non-TBI chemo-conditioning ( Fludarabin/Thiotepa/Treosulfan or Fludarabin/Thiotepa/Busulfan).

Кого берут в протокол
  • Age at time of screening less than 18 years
  • Indication for allogeneic HSCT
  • Complete remission (CR) before SCT
  • Written consent of the parents (legal guardian) and, if necessary, the minor patient via “Informed Consent Form”
  • No pregnancy
  • ALL is no secondary malignancy
  • No previous HSCT
  • HSCT is performed in a study participating centre
Кого не берут в протокол
  • Patients who do not fulfil the inclusion criteria
  • Non Hodgkin-Lymphoma
  • ALL with extramedullary involvement with indication for TBI
  • CNS involvement at screening timepoint
  • Trisomie 21
  • The whole protocol or essential parts are declined either by patient himself/herself or the respective legal guardian
  • No consent is given for saving and propagation of anonymous medical data for study reasons
  • Severe concomitant disease that does not allow treatment according to the protocol at the investigator’s discretion (e.g. malformation
  • syndromes, cardiac malformations, metabolic disorders)
  • Karnofsky / Lansky score < 50%
  • Subjects unwilling or unable to comply with the study procedures
Сколько пациентов должно пройти через исследование 1000 in 5 years
Status November 2013
EudraCT 2012-003032-22
Entry Study Register National Cancer Institute: Protocol ID NCT01949129
Руководитель протокола Christina Peters
С кем можно связаться

International Studynkoordinator

Prof. Dr. Christina Peters
St. Anna Kinderspital
Zimmermann Platz 10
A-1090 Wien
Telefon +43 (1) 40170 3100
Fax +43 (1) 40170 7430

Studiendokumenation und zentrales Datenoffice

Barbara Kristufek
St. Anna Kinderkrebsforschung e.V.,
Zimmermammplatz 10
1090 Wien
Telefon +43 (1) 40470 4755
Fax +43 (1) 40470 7430

Co-Sponsor Germany

Prof. Dr. med. Peter Bader
Klinikum der Johann-Wolfgang-Goethe-Universität
Zentrum für Kinder- und Jugendmedizin, Klinik III
Theodor-Stern-Kai 7
60590 Frankfurt
Telefon +49 (69) 6301 7542
Fax +49 (69) 6301 6700

Trial office Germany

Bettina Steinmetz
Klinikum der Johann Wolfgang Goethe-Universität
Pädiatrische Onkologie und Hämatologie
Theodor-Stern-Kai 7
60590 Frankfurt
Telefon +49 (69) 6301 85366
Fax +49 (69) 6301 4202

Участники исследования Austria, Australia, Belgium, Brazil, Canada, Czech Republic, Denmark, Finland, France, Germany, Hungary, Israel, Italy, Jordan, Netherlands, New Zealand, Norway, Saudi Arabia, Slovakia, Sweden, Switzerland, Turkey, Great Britain
Weitere Informationen Sponsor: St. Anna Kinderspital
Внутренние ссылки Studienliteratur SCT