Dataset Information

Avatrombopag for the treatment of thrombocytopenia post hematopoietic stem-cell transplantation.

ABSTRACT:

Background

Thrombocytopenia post hematopoietic stem-cell transplantation (HCT) usually contributes to poor outcomes with no standardized treatment. Eltrombopag and romiplostim can be feasible for post-HCT thrombocytopenia, but the use of avatrombopag has not yet been evaluated.

Objectives

We aimed to evaluate the efficacy and safety of avatrombopag treatment in patients diagnosed with post-HCT thrombocytopenia.

Design

In this retrospective study, we evaluated the efficacy and safety of avatrombopag treatment in a cohort of 61 patients diagnosed with thrombocytopenia post HCT in our clinical center.

Methods

Avatrombopag was initiated at 20 mg daily, with a dosage adjustment to achieve platelet recovery to >20 × 10⁹/l independent from transfusion for 7 consecutive days (overall response, OR) or to >50 × 10⁹/l free from transfusion for 7 consecutive days (complete response, CR). Factors influencing OR and CR were studied in univariate and multivariate analyses, respectively. Within the follow-up, adverse events like myelofibrosis, thrombosis, and organ toxicities were monitored carefully.

Results

The overall response rate (ORR) to avatrombopag was 68.9% and the cumulative incidence (CI) of OR was 69.1%. The complete response rate (CRR) and the CI of CR were both 39.3%. The median days from avatrombopag initiation to OR and CR were 21 and 25 days, respectively. An adequate number of megakaryocytes before the initiation of avatrombopag was an independent protective factor of avatrombopag treatment for OR (hazard ratio, HR = 4.628, 95% confidence interval 1.92-11.15, p = 0.0006) and CR (HR = 4.892, 95% confidence interval 1.58-15.18, p = 0.006). Avatrombopag was well tolerated in all patients with no severe adverse events.

Conclusion

Our findings suggested that avatrombopag can be optional for thrombocytopenia post HCT.

SUBMITTER: Zhou M

PROVIDER: S-EPMC9523859 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature

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Publications

Avatrombopag for the treatment of thrombocytopenia post hematopoietic stem-cell transplantation.

Zhou Meng M Qi Jiaqian J Gu Chengyuan C Wang Hong H Zhang Ziyan Z Wu Depei D Han Yue Y

Therapeutic advances in hematology 20220928

<h4>Background</h4>Thrombocytopenia post hematopoietic stem-cell transplantation (HCT) usually contributes to poor outcomes with no standardized treatment. Eltrombopag and romiplostim can be feasible for post-HCT thrombocytopenia, but the use of avatrombopag has not yet been evaluated.<h4>Objectives</h4>We aimed to evaluate the efficacy and safety of avatrombopag treatment in patients diagnosed with post-HCT thrombocytopenia.<h4>Design</h4>In this retrospective study, we evaluated the efficacy a ...[more]

PMID: 36185780

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Project description:BackgroundHematopoietic stem cell transplantation (HSCT) is a standard therapy strategy for most malignant disorders in children. However, transplant-related pneumonia remains a major therapy challenge and mesenchymal stromal cells (MSCs) are rarely reported in HSCT-related pneumonia. The aim of our study was to assess the efficacy of MSC for HSCT-related pneumonia in children.MethodsWe retrospectively retrieved HSCT-related (severe and non-severe) pneumonia cases (aged < 18 years), which underwent MSC treatment (MSC group) or non-MSC treatment (non-MSC group) in Guangzhou Women and Children's Medical Center, from December 2017 to December 2019. We investigated outcomes of the two different treatments among severe cases and non-severe cases, respectively. The primary endpoints were differences in overall cure rate and time to cure between MSC and non-MSC groups. The secondary endpoints were 180-day overall survival and cumulative cure rate.ResultsFinally, 31 severe pneumonia cases (16 in MSC group, 15 in non-MSC group) and 76 non-severe cases (31 in MSC group, 45 in non-MSC group) were enrolled in this study. Among severe pneumonia cases, overall cure rate in MSC group was significant higher than that in non-MSC group (12[75.0%] vs. 5[33.3%]; OR = 6.00, 95% CI [1.26-28.5]; p = 0.020); the time (days) to cure in MSC group was dramatically reduced compared with that in non-MSC group (36 [19-52] vs. 62 [42-81]; OR = 0.32, 95% CI [0.12-0.88]; p = 0.009); the 180-day overall survival in MSC group was better than that in non-MSC group (74.5% [45.4-89.6] vs. 33.3% [12.2-56.4]; p = 0.013). Among non-severe pneumonia cases, the time (days) to cure in MSC group was notably decreased compared with that in non-MSC group (28 [24-31] vs. 33 [26-39]; OR = 0.31, 95% CI [0.18-0.56]; p = 0.003). Compared with non-MSC group, MSC-treated patients achieved significant improvements of cumulative cure rate not only in severe pneumonia cases (p = 0.027), but also in non-severe cases (p < 0.001).ConclusionsThis study revealed that MSC treatment could contribute to improving outcomes in children with pneumonia post-HSCT, especially in severe cases. These findings suggest MSC treatment as a promising therapy for HSCT-related pneumonia in children.

Dataset Information

Avatrombopag for the treatment of thrombocytopenia post hematopoietic stem-cell transplantation.

Background

Objectives

Design

Methods

Results

Conclusion

Publications

Avatrombopag for the treatment of thrombocytopenia post hematopoietic stem-cell transplantation.

Similar Datasets

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