EBMT NEWSLETTER | June 2017 | Volume 57 - Issue 3

Important dates

RACE study
Allogeneic HSCT in acquired Pure Red Cell Aplasia
Data Quality Initiative of the SAAWP
Use of Eltrombopag in Aplastic Anaemia
CML transplanted in active blast crisis
CGD transplant outcome (update)
More News: CALM Study

RACE study

A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients.

Study design: Open label randomised clinical trial
Phase: III
Primary objective: To investigate whether Eltrombopag added to standard immunosuppressive treatment increases the rate of early (at three months) complete response in untreated aplastic anemia patients.

Status: June 9, 2017
Countries # sites # open sites # enrolling sites # patients
France 8 7 7 47
Germany 5 0 0 0
Italy 9 3 1 7
The Netherlands 4 4 2 14
Spain 6 2 1 2
Switzerland 3 1 1 5
United Kindgdom 5 4 3 19
TOTAL 40 21 15 94

Allogeneic HSCT in acquired Pure Red Cell Aplasia

A study by the SAAWP

***Final call for data!***

On June 1st,  a final request has been sent to centres with patients receiving an allogeneic transplant for acquired Pure Red Cell Aplasia between 2000 and 2015. Transplants in this disease are rare and we hope to receive all data which is available.

Please send in your patient data using the prefilled form(s) before June 26th 2017 to the EBMT Data Office in Leiden via saawpebmt@lumc.nl

Data Quality Initiative of the SAAWP

This year we started a Data Quality Initiative in the SAAWP to improve the quality of the data already available in the EBMT registry database. In March 2017 Excel files with ProMISe data have been sent to more than 300 centres. We are very grateful for the files that have been completed and returned thus far! We realise that completing the Excel file requires an investment of your time and manpower. So why help us out?
  • At least 5 new proposals as well as many future studies will use the data made available by these initiatives.
  • Principal investigators from centres that contributed their patient data will be considered as co-author of the resulting publications.
  • In addition to increasing the scientific output, we anticipate that the future workload regarding the provision of missing data will be reduced.
Therefore we want to encourage you to participate in this initiative and to return the Excel file.

If you have questions, please contact Cora Knol of the EBMT Data Office via saawpebmt@lumc.nl.

Use of Eltrombopag in Aplastic Anaemia

A study by the SAAWP

***Final call for data!***

The Severe Aplastic Anemia Working Party of the EBMT is interested in understanding the use of Eltrombopag in Europe. The aim of this study is to investigate the safety and efficacy of Eltrombopag in patients with SAA receiving the drug for initial immunosuppressive treatment or for incomplete response or to treat relapse.
Inclusion criteria for this study are:
  • Patient diagnosed with SAA, vSAA, mSAA or PNH with AA phenotype, and
  • Treated with thrombopoetin agonists (Eltrombopag, Romiplostim) as part of either:
    • The first treatment (outside of EBMT studies; e.g. RACE or EMAA)
    • A rescue treatment
Data capture concerns line of treatment, indication, response to treatments as well as treatment start and stop dates. To facilitate data management we have limited data collection to a short MED-C form that should be easy to fill out. If you have eligible patient(s) please return the completed form by July 30th 2017.
For more information or to request the MED-C form, please contact Cora Knol/Paul Bosman via saawpebmt@lumc.nl

CML transplanted in active blast crisis

***Data request***

PI: Aleksandar Radujkovic

The EBMT Chronic Malignancy Working Party (CMWP) would like to invite you to participate in a retrospective study on “Allogeneic stem cell transplantation for blast crisis CML in the era of tyrosine kinase inhibitors”, focusing on the identification of pretransplant prognostic factors.
As this is a relevant topic with very little published data we look forward to your participation. If you’re interested to participate in the data request, please contact Henric-Jan Blok: h.j.p.blok@lumc.nl

CGD transplant outcome (update)

***Data request***

PI: Robert Chiesa

Allogeneic haematopoietic stem cell transplantation (HSCT) has been increasingly used to treat patients with Chronic Granulomatous Disease (CGD).
Nevertheless the available data on the outcome of patients with CGD after HSCT is still limited. In fact, the last EBMT retrospective study on this subject described only 27 patients (Seger et al, Blood 2002), while over the last 15 years more than 400 patients with CGD have been transplanted.
We have the opportunity to update the results on HCT in CGD and to better define important prognostic factors, such as the role of conditioning regimen, stem cell donor and stem cell source.

If you’re interested to participate in the data request, please contact Henric-Jan Blok: h.j.p.blok@lumc.nl

More News:

CALM Study

CALM invoices can be send till 1st July 2017 (hard deadline)

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