A Non-Interventional Prospective Study on Recipient Pre-transplant Thymic Function as a Biomarker of Transplant Outcome after Allo-HSCT given for Hematological Malignancies.
*** this study is recruiting***
T-cell immune reconstitution is a critical parameter for transplant outcome. It is dependent on the thymic function, especially in children and young adults. Thymic function can be monitored using a non-invasive biological test called “TREC” (“T-cell Receptor Excision Circles”).Erica Knödler Antoine Toubert Andrea Velardi Christina Peters
Pilot studies indicated that the recipient thymic function before transplantation could be associated with the relapse risk and graft outcome. The goal of this EBMT study would be to assess if recipient pre-transplant TREC could be a useful prognostic biomarker in allo-HSCT.
The primary aim of this Non-Interventional Prospective study is to evaluate the impact of pretransplant thymic function on disease relapse and relapse-free survival in patients who undergo allo-HSCT in European Centers. To the end, the goal of the study would be to assess if recipient pre-transplant TREC could be a useful prognostic biomarker in allo-HSCT.
The secondary aims are to assess the impact of primary malignancy (T vs B ALL vs AML), and disease status at diagnosis on thymic function and the impact of recipient pretransplant thymic function on other outcome parameters (acute and chronic GVHD, survival, severe infections).
Hence, the Immunobiology Working Party (IWP) within the EBMT has developed this non-interventional prospective study on the role of recipient pre-transplant thymic function on outcome after Allo-HSCT given for hematological malignancies. To this end, we would need DNA samples (3 micrograms) stored before transplantation in patients affected by acute leukemia (ALL and AML). DNA stored for HLA typing could be used according to ethical and regulatory rules (TREC analysis is not a genetic test). We plan to include different allo-HSCT settings (genoidentical, MUD, CBT, Haplo-HSCT), conditioning regimen (myeloablative vs. non-myeloablative).
We expect to recruit an approximate total of up to 800 patients within two years of starting the study. Therefore, given the 2-year follow-up, the total study time frame is predicted to be 4 years. For this study the IWP limited the required information to a minimum. Clinical outcomes as defined under Primary and Secondary Aims will be assessed at 12, and 24 months after transplant. Patients should consent to have data submitted to EBMT before participating in the study. Patients withholding consent will be excluded.
When interested in participation, please contact Erica Knödler of the EBMT Data Office via firstname.lastname@example.org before February 1st, 2014.
We want to thank you in advance for your cooperation in this important study and are looking forward to your participation.
Data Office IWP Principal investigator Chair IWP Chair PDWP
Combined Studies - IWP
Call for data
We are witnessing an unusual shift in stem cell donor selection; after two decades during which the choice for an allogeneic donor for a patient without a HLA identical sibling donor was limited to an unrelated adult donor or Cord Blood Unit, more and more Transplantation Centres report surprisingly good results obtained by the transplantation of haploidentical family donors. Little is yet known on the long term outcome of the patients which are receiving these haploidentical transplants now and the impact of secondary factors such as immune reconstitution and IPA and NIMA immunity and regulation on long term outcome.
Fortunately the EBMT Immunobiology Working Party had the foresight to activate a retrospective study of over 600 haematopoietic haploidentical transplants performed between January 1995 and June 2012 which can tell us to which extent these two variables can influence long term outcome.
We hope that you can agree with us that this an unique opportunity to collect and analyse this unique data set and hope that you will support this important project. Obviously all individuals contributing information will be recognised as co-author.
All participating centres have received an email to on how to contribute data. Please do so before March 31st.
The Effect of 2nd generation TKI on the outcome after allogeneic SCT for Patients with CML:
A Non-Interventional Prospective Study
Patient Inclusion closed October 1st, 2013
As the study has reached its recruitment target of 390 patients, it was decided to close the study for patient inclusion per 01 October 2013.
Participating centres were able to submit their eligible patients until 31 December 2013.
In the upcoming weeks, participating centres will receive an overview of the patients they have included in the study.
In 2014 we will shift our focus to the collection of study data of the patients in the study.
Call for data
The chart below shows that although most patients have been registered in the database, the submission of MED B/C and follow up data for this study can be improved. Therefore we want to ask all participants to update their patients in the study at the earliest convenience.
Data for this study includes: study specific MED B form + Day100 form, short MED C form, yearly follow up form.
For study documents or information about the study, please contact Jennifer Hoek, study coordinator at the EBMT Data Office in Leiden, via email@example.com
Please, do not forget to send in your patients’ MED C form!
We would like to thank all participants for their ongoing cooperation in this important study!
CML subcommittee of the CMWP.
Non-interventional Prospective Study on the Role of Donor vs Recipient NK Cell Allo-reactivity in Haploidentical Hematopoietic Transplantation for Hematological Malignancies
*** this study is open for recruitment***
The Immunobiology Working Party (IWP) is conducting a study to evaluate the role of donor vs recipient NK cell allo-reactivity in haploidentical hematopoietic transplantation for hematologic malignancies. Donor-versus-recipient NK cell allo-reactivity is a key therapeutic element in the success of HLA haplotype mismatched (“haploidentical”) hematopoietic stem cell transplants for acute myeloid leukemia. The role of NK cell allo-reactivity will be assessed separately in T cell depleted and T cell replete transplants in the following two patient populations: 1) AML or ALL in any remission; 2) AML or ALL in chemo-resistant relapse.
Please see the leaflet and invitation letter for more information on recruitment for this study.
Final call for data [deadline postponed till February 1, 2014]
The retrospective study on Severe Congenital Neutropenia (SCN)
The definitive cure for SCN is HSCT, but data on its outcome are derived from small series and case reports in the past. Three EBMT Working Parties (SAA, Inborn Errors and Pediatric Diseases) together are conducting a retrospective study to improve knowledge about this topic. The aim of the study is to collect extensive data on SCN patients who underwent HSCT, to analyze the outcome of the procedure and to identify its associated risk factors.
In 2013, a feasibility check showed that of the 62 centres with SCN patients (n=119), 27 centres agreed to participate, 5 were unable to comply and of 30 centres we did not yet receive a reply. Twenty of the participating centres returned the disease specific form C (n=46).
A very preliminary analysis showed a significant 5-years OS in patients transplanted before the age of 10 (p= 0,046). Also, the 5-years OS for patients transplanted after the year 2000 was better than before 2000.
In order to collect as optimal data as possible of the 119 SCN patients, in October 2013 a (MEDB/C) prefilled questionnaire was sent to the centres. And this request was followed by a reminder in December/January.
We would greatly appreciate to receive the prefilled forms back at the EBMT Leiden Data Office by February 1, 2014. We expect to then start with the final analysis for the manuscript and publication of this unique and important study.
Please do not hesitate to get in touch with the Leiden Data Office (SAAWPebmt@LUMC.NL) in case of any questions or if you would like to receive the prefilled forms (perhaps in another format).
The principal investigators are grateful to all participating centres and they hope to provide the first solid evidence that HSCT in SCN either from a related or unrelated donor is a valid option for non/low responders to G-CSF SCN patients or for those transforming into MDS/acute leukemia.
The CALM study
Collaboration to Collect Autologous transplant outcomes in Lymphoma and Myeloma patients
Call for data!!
First we would like to thank you all for sending in your data and for the hard work you have done! Almost all CALM centres are up and running and we have received a lot of data these last few months. Of the 9500 transplants we are collecting in total, 4600 MED-B and 4900 MED-C forms have been received. We are well on our way but still need more data in order to reach our target of 9500 transplants.
Therefore we would still like to urge you to please send in your MED B, AUTOgraft and MED C data as soon as possible, if you haven’t already done so. We want to enter all data in ProMISe before Remedy goes live.
If you are not performing your own data entry, you are asked to send the MED-B data for the prospective transplantations (not yet registered in ProMISe) to the EBMT Data Office in London.
The MED-B data for the retrospective transplantations (already registered in ProMISe) as well as the MED-C data for all transplantations should be sent to the EBMT Data Office in Leiden. Please note that for MM for the consecutive 2nd and 3rd transplants a separate MED C from should be completed.
If you are performing your own data entry, please be reminded to create a working party record in ProMISe and enter the CALM study code 42206644. If you have already entered your patients in Promise please let us know and we can assign the CALM study code for you.
In order to spread the workload, we would like to ask you to send us data on an on-going base.
Extension data submission deadline:
The deadline for MED B/C data submission has been extended until February 28, 2014.
Data quality Excel files
The last months data quality files have been send out to ask for the baseline MED B and MED C items that are missing. If your centre has received such a file, please return this file before February 15, 2014. In March a similar file will be created for the follow-up items on the MED B form.
These weeks you will receive an overview with the available follow-up years in ProMISe. The aim is to have a follow-up form for each year following transplant until 2014 or the death of the patient. Please have a look at these overviews and complete the missing forms.
CALM study coordination
As of December 01, 2013 until April 01, 2014, Steffie van der Werf is on maternity leave. Any CALM study related correspondence/questions can be send to Paul Bosman or Cora Knol via e-mail address: firstname.lastname@example.org.Cora Knol
Invitation to participate in the non-interventional prospective study on the resistance pattern of Gram-negative bacteria isolated from blood from HSCT recipients.
There is a significant increase in resistant bacteria emerging in HSCT recipients. These resistant bacteria may be associated with increased mortality and the treatment options are limited. To provide the currently best empirical coverage and to control the growing resistance, knowledge of trends in antibiotic susceptibility as well as risk factors is essential. For this reason we would like to perform a non-interventional prospective multicentre study among EBMT centres.
Within 1 year we aim to collect 365 episodes of Gram-negative bacterial infections by following 3650 transplants recipients. The background information about the transplantations will be collected via the standard MED A form. Data on all the episodes of Gram-negative bacteria blood stream infections will be collected prospectively from the initiation of the conditioning treatment until the end of the first 6 months after the HSCT (or death or lost follow-up, if they occur earlier). Data on the patients who will develop a Gram-negative infection will be reported using a special MED C form and includes data on the pathogen and antimicrobial susceptibility of the pathogen, the clinical findings during the infection, presence of certain risk factors at the time of infection, treatment and outcome. The responsibility of centers participating in this study will be to fill the computerised short MED C form within 3 months of the Gram-negative bacteremia episode (it takes less than 30 minutes to complete it).
Study period: February 2014 – July 2015.
When your centre is interested in participating in this important study, or when you have any questions or concerns, please contact Jennifer Hoek of the EBMT Data Office via email@example.com .
Thanking you in advance for your collaboration,
Dina Averbuch Dan Engelhard Simone Cesaro
Study Investigator Study Investigator IDWP Chair