EBMT NEWSLETTER | November 2016 | Volume 54 - Issue 5

EBMT
Important dates
Content:
Prospective clinical trials
Current Treatment of HCV Infection after HSCT
IPAT: Impact of Pre-existing invasive Aspergillosis on allogeneic stem cell Transplantation
Pneumocystis Jirovecii Pneumonia (PcP) after Allogeneic HSCT
‘Domino’ HSCT – Indications, Treatment Strategy and Outcome

Prospective clinical trials

The EBMT Clinical Trials Office is currently working on the RACE trial and the older trials that are in long-term follow-up.

RACE trial 

Principal Investigators: Regis Peffault de Latour, Antonio M Risitano
Clinical Trial Coordinator: Ruzena Uddin
  • 17 patients since the summer in this rare disease
  • 1st Spanish patient enrolled
  • A total of 49 patients recruited from 16 open centres
  • The countries open for recruitment are France, Italy, Netherlands, Spain and the UK
  • Switzerland will be open soon; Germany is temporarily on hold

CMV Prophylaxis trial

EBMT Steering Group: Per Ljungman, Rafael Duarte
Sponsor: Merck
Clinical Trial Coordinator: Sue Philpott
  • This phase III randomised trial (Letermovir for CMV prophylaxis) has met its primary endpoint
  • EBMT assisted Merck on this trial who issued a press release on October 19th 
  • The Merck-EBMT collaboration was very successful

Current Treatment of HCV Infection after HSCT

IDWP non-interventional prospective study

*** Recruiting ***
 
PI: Malgorzata Mikulska
 
When you have a patient with HCV infection, please contact the IDWP Data Office via IDWPebmt@LUMC.nl

The availability of novel therapies with oral directly acting antivirals might prompt clinicians caring for HCV-positive HSCT recipients to prescribe the treatment more frequently and possibly earlier after HSCT.

This study will focus on treatment strategies in HCV-positive HSCT recipients and might provide additional data compared to previous older cohorts in the area of non-invasive assessment of fibrosis, HCV-RNA levels and genotyping. The main focus is the rate of treatment, the combination of drugs chosen, the length of treatment and the outcome.

Inclusion criteria

All HCV-RNA positive patients  cared for between December 2015 and  November 2017, who underwent HSCT (auto or allo) from any time to May 2017 .

Data collection

Patients not on treatment: 1 baseline form, 1 follow-up form at end of study.
Patients on treatment: 1 treatment form, 1 follow-up form 6 months after the end of treatment .

IPAT: Impact of Pre-existing invasive Aspergillosis on allogeneic stem cell Transplantation

IDWP non-interventional prospective study

*** Recruiting***

PI: Olaf Penack

01 May 2016 - 31 October 2017

In patients with pre-existing invasive aspergillosis (IA) allo-SCT is feasible without progression of fungal infection. However, the influence of invasive mould infections on transplant related complications and on long term survival has not been investigated in a larger patient cohort under current conditions.

With this non-interventional prospective study we want to assess clinical outcome of patients with —and without— history of pre-existing  IA undergoing allo-HSCT, in terms of non-relapse mortality, overall mortality and fungal infectious morbidity.

Study population

First allo-HSCT in patients with acute leukaemia and MDS given stem cell grafts.
            Cohort 1: History of probable or proven invasive aspergillosis
            Cohort 2: No History of probable or proven invasive aspergillosis.

Study period

01 May 2016 - 31 October 2017, with one year follow up.

Data collection

All patients: new MED A; Patient Registration Form; MED C follow up forms.
Only for patients with proven/probable IA and additional form needs to be completed.
 
When interested in participation, please contact the IDWP Data Office via
IDWPebmt@LUMC.nl

Pneumocystis Jirovecii Pneumonia (PcP) after Allogeneic HSCT

IDWP non-interventional prospective study

 ***Recruiting***
 
PI: Christine Robin

01 March 2016 - 01 March 2017

When you have a patient with PcP infection, please contact the IDWP Data Office via IDWPebmt@LUMC.nl

We aim to collect 100 cases. The study is open to EBMT centres using BAL for the diagnosis of PcP. You may participate even when you have no pneumocystis PCR on site.

Study period

01 March 2016 – 28 February 2017 + 90 days follow up.

Inclusion criteria of the PcP cases:

  • Allogeneic HSCT within the previous 24 months
  • New case (first onset) of PcP documented  in a BAL fluid, whatever the positive diagnostic test (cytology or IF or PCR) and whatever the presentation and treatment
  • Any age
  • Pre-or post-transplant informed consent to enter the data in the EBMT registry.

Exclusion criteria of the PcP cases:

  • Autologous HSCT
  • Allogeneic HSCT recipient transplanted more than 24 months before the onset of PcP
  • Second episode of PcP since allogeneic HSCT (patients diagnosed with PcP before allogeneic HSCT are not excluded).
The study aims at identifying risk factors, timing and diagnostic methods of PcP after allogeneic HSCT within the EBMT. It consists of a prospective 12-months collection of new PcP cases occurring in the 24 months following an allogeneic HSCT. PcP is defined by any positive specific criteria in BAL (cytology or IF or PCR), whatever the clinical presentation and whether the patient has been treated or not. We will ask you for each PcP case to fill a specific short MedC. The IDWP data office will identify in your centre 2 control cases for one case of PcP and you will be asked to fill a short Med C for each of these controls. MED A registration of these patients is required.
The number of cases per EBMT centre during the last years is very low (0 up to 7 in very big centres) and this study represents few work for the centres, but may provide major information if most EBMT centres participate.

‘Domino’ HSCT – Indications, Treatment Strategy and Outcome

IEWP retrospective study

*** Recruiting ***

PI: Marc Bierings*
 
“Domino” or serial haematopoietic stem cell transplantation (HSCT) refers to a situation in which the donor of an allogeneic stem cell transplantation has previously received a haematopoietic stem cell transplantation as well. Several case reports have reported successful use of a domino HSCT in patients with severe combined immunodeficiency, suggesting that a domino donor may be a useful alternative to cord blood or adult donors. However, systematic investigation is needed to determine the efficacy domino-HSCT as a treatment modality.

For this reason the Inborn Errors Working Party of EBMT has created a short survey to identify domino transplants in EBMT centres. We invite you to complete the survey via the following link: https://www.surveymonkey.com/r/dominotransplant
 
When your centre has ever performed a domino transplant, you will be asked to complete a questionnaire  to document the case.
 
For more information, please contact the IEWP Data Office via iewpebmt@lumc.nl
 
*Marc Bierings and Mirjam Belderbos, Department of Paediatric Haematology and Stem Cell Transplantation, University Medical Centre Utrecht, Utrecht, The Netherlands
 
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