Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation
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 s...
Main Authors: | , , , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
SAGE Publishing
2020-10-01
|
Series: | Therapeutic Advances in Hematology |
Online Access: | https://doi.org/10.1177/2040620720961910 |
id |
doaj-591132220bad454a83cd9b71bca99cc5 |
---|---|
record_format |
Article |
spelling |
doaj-591132220bad454a83cd9b71bca99cc52020-11-25T03:08:41ZengSAGE PublishingTherapeutic Advances in Hematology2040-62152020-10-011110.1177/2040620720961910Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantationSemra AydinChiara DellacasaSara ManettaLuisa GiacconeLaura GodioGiorgia IovinoBenedetto BrunoAlessandro BuscaBackground: 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.https://doi.org/10.1177/2040620720961910 |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Semra Aydin Chiara Dellacasa Sara Manetta Luisa Giaccone Laura Godio Giorgia Iovino Benedetto Bruno Alessandro Busca |
spellingShingle |
Semra Aydin Chiara Dellacasa Sara Manetta Luisa Giaccone Laura Godio Giorgia Iovino Benedetto Bruno Alessandro Busca Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation Therapeutic Advances in Hematology |
author_facet |
Semra Aydin Chiara Dellacasa Sara Manetta Luisa Giaccone Laura Godio Giorgia Iovino Benedetto Bruno Alessandro Busca |
author_sort |
Semra Aydin |
title |
Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation |
title_short |
Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation |
title_full |
Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation |
title_fullStr |
Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation |
title_full_unstemmed |
Rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation |
title_sort |
rescue treatment with eltrombopag in refractory cytopenias after allogeneic stem cell transplantation |
publisher |
SAGE Publishing |
series |
Therapeutic Advances in Hematology |
issn |
2040-6215 |
publishDate |
2020-10-01 |
description |
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. |
url |
https://doi.org/10.1177/2040620720961910 |
work_keys_str_mv |
AT semraaydin rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT chiaradellacasa rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT saramanetta rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT luisagiaccone rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT lauragodio rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT giorgiaiovino rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT benedettobruno rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation AT alessandrobusca rescuetreatmentwitheltrombopaginrefractorycytopeniasafterallogeneicstemcelltransplantation |
_version_ |
1724664781918437376 |