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Role of Complement System in Human Allogeneic Haematopoietic Stem Cell Transplantation

18 Years
65 Years
Open (Enrolling)
Allografted With Myeloablative Conditioning

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Trial Information

Role of Complement System in Human Allogeneic Haematopoietic Stem Cell Transplantation

The study will be performed in allografted patients with myeloablative conditioning for an
haematological malignancy from 3 adult transplant units.

Patients will be followed for at least12 months after transplantation and blood samples
drawn before conditioning and once a week for 12 weeks after transplantation to analyze:

- serum concentration of Complement factors (C3, C4, B factor), Complement regulatory
proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of
Complement regulatory molecules such as CD46, CD55 and CD59.

- serum inflammatory cytokine levels

In addition, patients with clinical signs of gut GVHD will be explored by gastrointestinal
endoscopy to perform gut biopsies. C5b9 deposure will be then analyzed by
immunohistochemistry on GVHD lesions.

Activation of complement system will be defined by a decrease of complement factor levels of
50% and values under lower physiological limits. The clinical evolution and the inflammatory
cytokine profile of patients with such an activation profile will be compared to that of
those without complement activation.

A data base containing biological and clinical data will be established. Biological results
will be correlated to post-transplant clinical events, in particular the occurrence of gut
GVHD but also non relapse mortality and overall survival by adapted statistical tests
(comparison of percentages by Chi-2 of Pearson, comparison of survival curves by logrank,
multivariate analysis by logistic regression test or cox model).

The number of required patients will be established by comparison of the percentage of gut
GVHD in the patients with or without complement activation. Based on our preliminary
results, we hypothesize that 2/3 patients will not have complement activation among whose
20% will develop acute gut GVHD. We expect an increase of acute gut GVHD up to 60% of the
patients with complement activation that would represent 1/3 of the cohort.

With a bilateral alpha risk of 5% and a power of 80%, the number of required patients is 23
in the activated group and 46 in the non activated group, thus a total of 69 patients.

Inclusion Criteria:

- Allografted patients with myeloablative conditioning for an haematological malignancy

- Age > 18 years old and < 65 years.

- The patient must have access to social insurance according to local regulations.

- Patient must give a written informed consent (personally signed and dated) before
completing any study related procedure

Exclusion Criteria:

- Age < 18 years old and > 65 years

- Patient with active infection HIV, HTLV1, Hepatite B ou C

- Uncontrolled infection(s), (i.e. documented bacterial, parasitical, or fungal

- Patient with lupus

- Patient with transaminases > 5N, TP<30% with Facteur V < 30% before allogreffe

- Creatinine clearance < 50ml/min

- Absence of any psychological condition potentially hampering signing informed consent

- Patient refused to sign informed consent

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Outcome Measure:

Activation of the complement system and the development of acute gut GvHD

Outcome Description:

Assessment of the activation of the complement system after human allogeneic stem cell transplantation and of its potential correlation with the development of acute gut GvHD

Outcome Time Frame:

12 weeks

Safety Issue:


Principal Investigator

Marie-Thérèse RUBIO, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Saint Antoine Hospital


France: Ministry of Health

Study ID:

CRC 08025



Start Date:

April 2010

Completion Date:

July 2013

Related Keywords:

  • Allografted With Myeloablative Conditioning
  • Allogeneic haematopoietic stem cell transplantation (HSCT)
  • Myeloablative conditioning for an haematological malignancy
  • Graft-versus-host disease (GvHD)
  • Activation of complement system
  • Gut GVHD
  • Graft vs Host Disease