# Effective treatment with daratumumab in post-HSCT refractory immune-mediated cytopenias: a case report and literature review

**Authors:** Xiao-yu Jing, Dong-jun Li, Sheng-nan Su, Qing-kai Dai, Shu-wen Sun, Liang Huang, Yuan Ai, Ju Gao, Yi-Ping Zhu, Jia-qi Ni, Xiao-xi Lu

PMC · DOI: 10.3389/fimmu.2025.1625365 · Frontiers in Immunology · 2025-08-01

## TL;DR

A child with severe immune-related blood disorders after a stem cell transplant showed improvement with daratumumab treatment.

## Contribution

Daratumumab is proposed as a novel treatment for refractory immune-mediated cytopenias after HSCT.

## Key findings

- Daratumumab induced a rapid and sustained response in a refractory immune-mediated cytopenia case.
- Platelet levels normalized after re-initiating daratumumab therapy.
- The only significant adverse effect was delayed recovery of humoral immunity.

## Abstract

Immune-mediated cytopenias (IMCs) following allogeneic hematopoietic stem cell transplantation (HSCT) can lead to substantial morbidity and mortality, presenting a major therapeutic obstacle. Here, we report a case of a pediatric patient with acquired aplastic anemia. Nine months after HSCT, this patient developed severe, refractory hemolytic anemia and immune-mediated thrombocytopenia (IMT). Despite treatment with corticosteroids, intravenous immunoglobulin (IVIG), rituximab, along with avatrombopag, romiplostim, acetylcysteine, and decitabine, the patient’s platelet count showed no signs of improvement. Subsequently, daratumumab, a monoclonal antibody targeting CD38, was administered. This treatment induced a rapid and sustained response. Four months after initial daratumumab administration, the percentage of CD38-positive immune cells in the patient’s peripheral blood increased, which was concurrent with another decline in platelet levels. After re-initiating daratumumab therapy, the patient’s platelet count returned to normal levels. The only significant adverse effect noted was a delayed recovery of humoral immunity. Daratumumab, by targeting antibody-producing plasma cells, shows promise as a therapeutic alternative for refractory IMCs in post-HSCT patients.

## Linked entities

- **Proteins:** CD38 (CD38 molecule)
- **Chemicals:** avatrombopag (PubChem CID 9852519), acetylcysteine (PubChem CID 12035), decitabine (PubChem CID 451668)
- **Diseases:** acquired aplastic anemia (MONDO:0015610), hemolytic anemia (MONDO:0003664)
- **Species:** Homo sapiens (taxon 9606)

## Full-text entities

- **Genes:** CD38 (CD38 molecule) [NCBI Gene 952] {aka ADPRC 1, ADPRC1, cADPR1}
- **Diseases:** hemolytic anemia (MESH:D000743), IMCs (MESH:C567355), IMT (MESH:D016553), aplastic anemia (MESH:D000741)
- **Chemicals:** decitabine (MESH:D000077209), avatrombopag (MESH:C533238), Daratumumab (MESH:C556306), rituximab (MESH:D000069283), acetylcysteine (MESH:D000111)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## Figures

3 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12353738/full.md

## References

47 references — full list in the complete paper: https://tomesphere.com/paper/PMC12353738/full.md

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Source: https://tomesphere.com/paper/PMC12353738