Use of Thrombopoietin Receptor Agonists in Prolonged Thrombocytopenia after Hematopoietic Stem Cell Transplantation

Mahat, Upendra; Rotz, Seth J.; Hanna, Rabi

doi:10.1016/j.bbmt.2019.12.003

Cited by 45 publications

(43 citation statements)

References 82 publications

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“…Most studies have analyzed its use in the treatment of post-HSCT thrombocytopenia in adults and have reported response rates between 57% and 72% and a favorable toxicity profile. [16][17][18][19][20] In children in the same context, data are limited to scarce case reports. [21][22][23] To our knowledge, there have been no studies on the use of eltrombopag to treat PGF post-HSCT in children.…”

Section: Discussionmentioning

confidence: 99%

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Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children

Uria-Oficialdegui

Alonso

Benítez‐Carabante

et al. 2021

Pediatric Transplantation

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Poor graft function (PGF) is a relatively common complication of hematopoietic stem cell transplantation (HSCT), with an incidence of 5% to 27% depending on the series. [1][2][3][4][5] PGF is characterized by peripheral cytopenia affecting two or three hematopoietic lineages associated with decreased bone marrow cellularity and complete donor chimerism (>95%).Factors associated with PGF are diverse: underlying disease and disease status at the time of transplant, donor type, HLA antibodies against HLA donor antigens, graft cell content, post-HSCT infections, myelotoxic drugs, graft-vs-host disease (GVHD), iron overload and splenomegaly. 1,6 There are few treatments available. 5 Current options include blood transfusions, use of G-CSF, CD34 + -selected stem cell boosts (SCB) infusions, 2,5 and in experimental settings, mesenchymal cells

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Section: Discussionmentioning

confidence: 99%

“…However, experience with eltrombopag in PGF post‐HCST is limited. Most studies have analyzed its use in the treatment of post‐HSCT thrombocytopenia in adults and have reported response rates between 57% and 72% and a favorable toxicity profile 16‐20 . In children in the same context, data are limited to scarce case reports 21‐23 …”

Section: Discussionmentioning

confidence: 99%

Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children

Uria-Oficialdegui

Alonso

Benítez‐Carabante

et al. 2021

Pediatric Transplantation

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“… 169 Response, defined as platelet count >50 × 10 9 /L free of platelet transfusion, was achieved in 18 patients at a median (interquartile range [IQR]) time of 45 (29–41) days (range, 21–77 days), with a median (IQR) dose of 5 (4–6.8) µg/kg (range, 1–11 µg/kg); such a response was sustained in 16/18 patients for ≥8 consecutive weeks, independent of platelet transfusions. 169 Mahat et al 170 reviewed 12 studies (six case series and six case reports) involving the use of romiplostim for prolonged post-HSCT thrombocytopenia (primary thrombocytopenia in 17 patients and secondary in 32 patients); a platelet response of >50 × 10 9 /L free of platelet transfusion was observed in 40 out of the 49 patients (82%) overall.…”

Section: Clinical Evidence and Rationale For Use Of Romiplostim Beyond Itpmentioning

confidence: 99%

A Review of Romiplostim Mechanism of Action and Clinical Applicability

Bussel¹,

Soff²,

Balduzzi³

et al. 2021

DDDT

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Thrombocytopenia results from a variety of conditions, including radiation, chemotherapy, autoimmune disease, bone marrow disorders, pathologic conditions associated with surgical procedures, hematopoietic stem cell transplant (HSCT), and hematologic disorders associated with severe aplastic anemia. Immune thrombocytopenia (ITP) is caused by immune reactions that accelerate destruction and reduce production of platelets. Thrombopoietin (TPO) is a critical component of platelet production pathways, and TPO receptor agonists (TPO-RAs) are important for the management of ITP by increasing platelet production and reducing the need for other treatments. Romiplostim is a TPO-RA approved for use in patients with ITP in the United States, European Union, Australia, and several countries in Africa and Asia, as well as for use in patients with refractory aplastic anemia in Japan and Korea. Romiplostim binds to and activates the TPO receptor on megakaryocyte precursors, thus promoting cell proliferation and viability, resulting in increased platelet production. Through this mechanism, romiplostim reduces the need for other treatments and decreases bleeding events in patients with thrombocytopenia. In addition to its efficacy in ITP, studies have shown that romiplostim is effective in improving platelet counts in various settings, thereby highlighting the versatility of romiplostim. The efficacy of romiplostim in such disorders is currently under investigation. Here, we review the structure, mechanism, pharmacokinetics, and pharmacodynamics of romiplostim. We also summarize the clinical evidence supporting its use in ITP and other disorders that involve thrombocytopenia, including chemotherapy-induced thrombocytopenia, aplastic anemia, acute radiation syndrome, perisurgical thrombocytopenia, post-HSCT thrombocytopenia, and liver disease.

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“…In the setting of HSCT, several authors investigated retrospectively the role of eltrombopag in refractory thrombocytopenia after allogeneic HSCT and reported promising results with significant platelet recovery until achieving transfusion independence. 16 – 19 On the basis of these results current phase II trials using TPO agonists for prolonged thrombocytopenia after HSCT have been initiated, (NCT01791101, NCT01000051, NCT01927731).…”

Section: Introductionmentioning

confidence: 99%

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

Aydin

Dellacasa²,

Manetta³

et al. 2020

Therapeutic Advances in Hematology

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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.

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Use of Thrombopoietin Receptor Agonists in Prolonged Thrombocytopenia after Hematopoietic Stem Cell Transplantation

Cited by 45 publications

References 82 publications

Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children

Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children

A Review of Romiplostim Mechanism of Action and Clinical Applicability

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

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