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Open research questions in Hematopoietic Stem Cell Transplantation

25 unresolved questions extracted from the limitations and future-work sections of 264 Hematopoietic Stem Cell Transplantation papers in our library. Each links back to the study that raised it.

What the literature leaves open

  • We further recognize that cost-effectiveness and implementation immune monitoring, to advanced barriers availability of second-line agents for steroid-refractory disease) vary widely across clinical settings, and these factors must be weighed alongside risk profiles in shared decision-making. Prospective studies integrating these risk factors into a simple, cost-conscious risk scoring tool are warranted to translate these findings into actionable, universally applicable clinical algorithms.

    Clinical characteristics and risk factors of acute cutaneous graft-vs.-host disease following allogeneic hematopoietic stem cell transplantation in pediatric acute myeloid leukemia: a single-center retrospective study · 2026 · DOI

  • To reconcile these contradictory findings, we analyzed the effects of Septin7 deletion in hematopoietic cells from mice with either pan-hematopoietic or lymphoid lineage-specific Septin7 deletion.

    Septin7 is essential in early hematopoiesis, but redundant at later stages · 2026 · DOI

  • Consequently, the present study represents the first investigation focused exclusively on pediatric FA patients and the first to employ a uniform non-TBI conditioning regimen across the entire cohort, thereby addressing a significant gap in the evidence. Published data on post-HSCT AKI specifically in FA remain scarce.

    Early, frequent, yet reversible: acute kidney injury after hematopoietic stem cell transplantation in pediatric Fanconi anemia and long-term renal outcomes · 2026 · DOI

  • Adult long-term hematopoietic stem cells (LT-HSCs) are classically defined by self-renewal, multilineage regenerative capacity, and relative quiescence, but how and when lifelong LT-HSCs are established during development remains unclear.

    Hoxb5+ fetal liver hematopoietic stem cells establish lifelong hematopoiesis and exhibit enhanced ITGA4-dependent engraftment · 2026 · DOI

  • ConclusionsAn explainable, externally validated GVHD prediction framework was developed using heterogeneous registry-derived datasets, with systematic characterization of calibration drift across multiple external cohorts, an analysis rarely reported in prior GVHD prediction literature.

    External Validation and Calibration Assessment of Explainable Machine Learning Models for GVHD Prediction After Allogeneic HSCT · 2026 · DOI

  • The bone marrow (BM) vascular network plays crucial roles in driving bone development and supporting hematopoiesis, yet the mechanisms governing its specialized architecture, particularly sinusoidal morphogenesis, remain inadequately characterized.

    Specification of bone marrow sinusoids requires TIE2-mediated positive feedback involving COUPTFII and VEGFR3 · 2026 · DOI

  • Transcriptomics also identified genes that are understudied in the context of oxygen dependency including MDM4 pathway and PRSS2 , which we found is an mRNA biomarker for hematopoietic cell potency.

    Local oxygen tension dictates hematopoietic cell growth and potency · 2026 · DOI

  • Conclusion Our results demonstrate that specific inhibition of BTK with acalabrutinib has a potentially beneficial role in ameliorating sclerodermatous cGVHD by improving the survival and clinical benefits and warrants further investigation in patients with cGVHD.

    Targeting Bruton tyrosine kinase with acalabrutinib attenuates murine sclerodermatous chronic graft versus host disease · 2026 · DOI

  • Safety-net hospitals (SNHs) cater to the medical needs of underserved populations, but their impact on the outcomes of hematopoietic stem cell transplantation (HSCT) remains unclear.

    Impact of hospital safety-net status on in-hospital and short-outcomes after hematopoietic stem cell transplantation · 2026 · DOI

  • This case is the first example in a man of haemato- poietic artificially induced chimerism of long duration: at the time of writing it had already lasted longer than eight months, following a conditioning that had been no different from that which had made possible similar grafting in various species of animals.6 16 In the light of the above-mentioned experiments in mice,8 the fact that the patient had had no previous blood trans- fusions was regarded as particularly important.

    Haematopoietic Chimera in Man After Allogenic (Homologous) Bone-marrow Transplantation · 1963 · DOI

  • This mechanism enables LADTs to restore alloreactivity only in lymphoid organs where B cells are abundant but not in other organs where B cells are scarce.

    Lymphoid Tissue-Activated Donor T cells (LADTs) for Improved Donor Lymphocyte Infusion (DLI) against Blood Cancers 2250463 · 2026 · DOI

  • This dual TPO-RA regimen warrants further investigation as a potential first-line option for HSCT-ineligible VSAA patients.

    Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy · 2026 · DOI

  • In spite of the fact that miniature inbred and domestic pig breeds have been used as large animal models in hemopoietic research and in experimental bone marrow transplantation, data concerning the proliferative rate of porcine progenitor cells are still lacking.

    Pig bone marrow and peripheral blood erythroid progenitor cells in S phase of the cell cycle · 2000

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25 open questions have been extracted from the limitations and future-work passages of 264 Hematopoietic Stem Cell Transplantation papers in our library. Each one below links back to the study that raised it, so you can read the original claim in context.

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