PHARMACOTHERAPY

Multipotent mesenchymal stromal cell therapy for steroid-refractory acute graft-versus-host disease after allogeneic hematopoietic stem cell transplantation

BJH - volume 7, issue 6, december 2016

S. Servais MD, PhD, C. Grégoire MD, prof. F. Baron , E. Willems MD, PhD, A. Briquet PhD, E. Baudoux MD, O. Delloye PhD, O. Giet PhD, C. Lechanteur PhD, Y. Beguin MD, PhD

Summary

Steroid-refractory acute graft-versus-host disease is a severe complication after allogeneic stem cell transplantation. So far, its treatment remains very challenging since the current therapies do not offer significant benefits. Among the most recent approaches, multipotent mesenchymal stromal cell-based therapy has attracted great interest over the past decade. Here, we briefly reviewed the current knowledges about the immunomodulatory properties of multipotent mesenchymal stromal cells as well as results of preclinical and clinical studies having assessed their efficacy to modulate steroid-refractory acute graft-versus-host disease.

(BELG J HEMATOL 2016;7(6):229–35)

Read more

Discontinuation of imatinib in Belgian patients with chronic myeloid leukaemia

BJH - volume 7, issue 5, october 2016

P. Mineur MD, C. Doyen MD, PhD, N. Straetmans MD, PhD, K. Van Eygen MD, D. Pranger MD, A. Bosly MD, PhD, M. André MD, PhD, T. Devos MD, PhD, L. Knoops MD, PhD, On behalf of the MPN Belgian Hematological Society subcommittee

Summary

This article describes the Belgian register of chronic myeloid leukaemia patients who have stopped their treatment with imatinib in conditions comparable to the French STIM trial results: 44% remained in major molecular response off therapy; relapses appear rapidly after stopping imatinib and are responsive when the treatment is resumed.

(BELG J HEMATOL 2016;7(5):184–6)

Read more

Improving care for critically ill haematological patients in the ICU: a modern perspective

BJH - volume 7, issue 3, june 2016

I. Moors MD, P. Depuydt MD, PhD, F. Offner MD, PhD, D. Benoit MD, PhD

Summary

Outcome of critically ill haematological patients in the intensive care unit has substantially improved during the past decades, with current estimates for intensive care unit survival of 70–75% and one-year survival of 40–45%. Based on new insights, the approach towards critically ill haematological patients is changing, with a focus on early recognition of deteriorating patients in the ward and early referral to the intensive care unit when necessary. Broad admission policies should become the standard, with regular re-assessment of the level of care administered, relative to survival expectations and burden for the patient and family. Close collaboration and communication between attending intensivists and referring haematologists with complementary skills is essential to provide good quality of care, be it either achieving short- and long-term survival and good quality of life, or timely withdrawal of aggressive therapy and institution of appropriate comfort care.

(BELG J HEMATOL 2016; 7(3):112–7)

Read more

Nanobodies and thrombotic thrombocytopenic purpura

BJH - volume 7, issue 2, april 2016

A. Rogiers MD, E. Porcher , D. Dierickx MD, PhD

Summary

Thrombotic thrombocytopenic purpura is a non-malignant but life-threatening haematological disorder caused by deficiency of ADAMTS13, a metalloproteinase cleaving ultra-large von Willebrand factor multimer. Urgent initiation of therapeutic plasma exchange, in most cases associated with corticosteroids, has dramatically increased outcome of patients presenting with thrombotic thrombocytopenic purpura. Recently a phase II trial with caplacizumab, a humanised nanobody binding to the A1 domain of von Willebrand factor, has shown promising results by reducing the number of plasma exchanges with an acceptable safety profile.

(BELG J HEMATOL 2016;7(2):79–81)

Read more

Ibrutinib and idelalisib, the B-cell receptor antagonists available for use in daily clinical practice

BJH - volume 6, issue 5, december 2015

A. Janssens MD, PhD

summary

The first-in-class Bruton’s tyrosine kinase and phosphatidylinositol-3-kinase delta inhibitors have demonstrated impressive clinical activity and tolerability in several B-cell malignancies, both as single agent or in combination with rituximab. As reimbursement of ibrutinib and idelalisib by the Belgian national public health insurance has been granted, this review describes mechanism of action, dosage and administration, efficacy and tolerability. Although both molecules show a very favourable toxicity profile, treating physicians and patients must be aware of some medical events of interest.

(BELG J HEMATOL 2015;6(5):216–24)

Read more

The emerging role of monoclonal antibodies in the treatment of multiple myeloma

BJH - volume 6, issue 5, december 2015

K. Fostier MD, R. Schots MD, PhD

summary

Monoclonal antibodies have a profound impact on the prognosis and survival of patients with haematological malignancies. In the treatment of multiple myeloma, until recently, results of monoclonal antibodies have been disappointing. The introduction of two novel classes of monoclonal antibodies holds great promise to change this. Daratumumab (and related antibodies) is a monoclonal antibody directed to CD38, an intriguing multifunctional surface protein abundantly expressed on malignant plasma cells and their precursors. Daratumumab displays impressive single agent activity in heavily pretreated multiple myeloma patients and due to its favourable safety profile, this molecule seems to be an excellent accessory companion to known anti-multiple myeloma regimens and also in monotherapy as a maintenance agent. Elotuzumab, to the contrary, is an anti-CS1 monoclonal antibody, which does not show any clinically relevant single agent activity, but when combined with other anti-multiple myeloma drugs appears to greatly enhance their efficacy and can even revert the refractory state to the agents. When these promising results are confirmed in phase III trials, immunotherapy can finally be incorporated in the treatment schedule of newly diagnosed and relapsed/refractory multiple myeloma patients.

(BELG J HEMATOL 2015;6(5):209–15)

Read more

New perspectives with PI3K inhibitors in B-cell malignant hemopathies

BJH - volume 6, issue 4, october 2015

D. Bron MD, PhD, M. Maerevoet MD

summary

Phosphoinositide 3-kinase inhibitors represent a new group of promising targeted therapies for malignant hemopathies and primarily lymphoproliferative disorders. This short report summarises recent knowledge on the mechanism of action, the rationale to use it in humans bearing malignant hemopathies and preliminary clinical trials’ data that led to the Food and Drug Administration approval of one of these compounds (idelalisib).

(BELG J HEMATOL 2015;6(4): 152–5)

Read more