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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:ImportanceRuxolitinib, a selective inhibitor of the Janus kinases 1/2 signaling pathway, has shown a significant response in steroid-refractory chronic graft-vs-host disease (SR-cGVHD), a major cause of morbidity and mortality in individuals who have undergone allogeneic hematopoietic stem cell transplantation (HSCT).ObjectivesTo investigate the clinical response to ruxolitinib in patients with SR-cGVHD after allogeneic HSCT and to evaluate its safety profile during the treatment course.Design, setting, and participantsThis single-center case series included 41 consecutive patients who were treated with ruxolitinib for SR-cGVHD after allogeneic HSCT between August 2017 and December 2019. Data were collected from each patient's medical record at the First Affiliated Hospital of Zhejiang University School of Medicine. Data analysis was conducted from March to May 2020.ExposureRuxolitinib.Main outcomes and measuresTreatment responses, factors associated with response, and adverse effects during ruxolitinib administration.FindingsOverall, 41 patients (median [range] age, 31 [17-56] years; 14 [34.1%] women) were treated with ruxolitinib and included in this study. A total of 15 patients (36.6%) had a complete remission, and 14 (34.1%) had a partial remission, with an overall response rate of 70.7% (29 patients; 95% CI, 56.2%-85.3%). Lung involvement (odds ratio, 0.112; 95% CI, 0.020-0.639; P = .01) and matched related donors (odds ratio, 0.149; 95% CI, 0.022-0.981; P = .048) were associated with less favorable treatment response. Major adverse events associated with ruxolitinib were cytopenias and infectious complications. The median (range) follow-up for this cohort was 14.9 (1.4-32.5) months. Prolonged survival was observed in patients with a male donor (P = .006), complete remission before transplantation (P = .02), baseline moderate cGVHD (P = .02), and skin cGVHD (P = .001).Conclusions and relevanceIn this small, single-site case series, ruxolitinib demonstrated a significant response in heavily pretreated patients with SR-cGVHD and a reasonably well-tolerated safety profile. The results add to the body of literature suggesting ruxolitinib as a promising treatment option in SR-cGVHD.

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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