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Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation.

ABSTRACT: Background:Patients with post-transplant cytopenias due to poor graft function or primary engraftment failure show poor prognosis with a high mortality rate mainly because of graft versus host disease (GVHD), infection and/or bleeding. Treatment options are scarce and a CD34+ stem cell boost or a second bone marrow transplantation may be required to restore adequate haematopoiesis. Methods:In the present study patients with primary engraftment failure (n?=?1) and refractory poor graft function (n?=?11) were treated with eltrombopag in a single centre. The reason for eltrombopag treatment was trilineage cytopenia in six patients, bilineage cytopenia in three patients and single lineage cytopenia in three patients. Eltrombopag was initiated at a median of 214 (range: 120-877) days after haematopoietic stem cell transplantation (HCST) and administered for a median time of 114 (range: 12 days to >490) days. In 8/12 patients eltrombopag was introduced at a dose of 75?mg/day and then increased to 150?mg/day after 1?week; 1 patient was given 50?mg eltrombopag per day, and 3 patients received 75?mg daily. Results:In 10/12 patients eltrombopag significantly enhanced blood count values and patients became transfusion independent. Once stable haematological response was obtained, treatment was tapered until final discontinuation in 9/10 responding patients. No grade 3 or 4 toxicities were observed. At time of last follow up, 3/12 patients were dead, 2 due to disease relapse, 1 due to GVHD and pneumonia. All patients except one maintained their complete response and remain transfusion independent at a median of 858 (range: 429-1119) days. Conclusion:These preliminary data confirm that eltrombopag is able to rescue multilineage haematopoiesis in patients with treatment-refractory cytopenias after allogeneic HSCT.

SUBMITTER: Aydin S

PROVIDER: S-EPMC7594218 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation.

Aydin Semra S Dellacasa Chiara C Manetta Sara S Giaccone Luisa L Godio Laura L Iovino Giorgia G Bruno Benedetto B Busca Alessandro A

Therapeutic advances in hematology 20201020

<h4>Background</h4>Patients with post-transplant cytopenias due to poor graft function or primary engraftment failure show poor prognosis with a high mortality rate mainly because of graft <i>versus</i> host disease (GVHD), infection and/or bleeding. Treatment options are scarce and a CD34+ stem cell boost or a second bone marrow transplantation may be required to restore adequate haematopoiesis.<h4>Methods</h4>In the present study patients with primary engraftment failure (<i>n</i> = 1) and ref ...[more]

PMID: 33194161

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Project description:Background & aimsDurable remissions of Crohn's Disease (CD) have followed myeloablative conditioning therapy and allogeneic marrow transplantation. For patients with treatment-refractory disease, we used reduced-intensity conditioning to minimize toxicity, marrow from donors with low Polygenic Risk Scores for CD as cell sources, and protracted immune suppression to lower the risk of graft-versus-host disease (GVHD). Our aim was to achieve durable CD remissions while minimizing transplant-related complications.MethodsDNA from patients and their HLA-matched unrelated donors was genotyped and Polygenic Risk Scores calculated. Donor marrow was infused following non-myeloablative conditioning. Patient symptoms and endoscopic findings were documented at intervals after transplant.ResultsWe screened 807 patients, 143 of whom met eligibility criteria; 2 patients received allografts. Patient 1 had multiple complications and died at day 332 from respiratory failure. Patient 2 had resolution of CD symptoms until day 178 when CD recurred, associated with persistent host chimerism in both peripheral blood and intestinal mucosa. Withdrawal of immune suppression was followed by dominant donor immune chimerism in peripheral blood and resolution of CD findings. Over time, mucosal T-cells became donor-dominant. At 5 years after allografting, Patient 2 remained off all medications but had mild symptoms related to a jejunal stricture that required stricturoplasty at 6 years. At 8 years, she remains stable off medications.ConclusionsThe kinetics of immunologic chimerism after allogeneic marrow transplantation for CD patients depends on the intensity of the conditioning regimen and the magnitude of immune suppression. One patient achieved durable improvement of her previously refractory CD only after establishing donor immunologic chimerism in intestinal mucosa. Her course provides proof-of-principal for allografting as a potential treatment for refractory CD, but an immunoablative conditioning regimen should be considered for future studies.(ClinicalTrials.gov, NCT01570348).

Dataset Information

Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation.

Publications

Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation.

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