STEM CELL transplantation using donor cells can offer meaningful long-term survival for patients with a rare and aggressive form of blood cancer.
New findings from the largest study of its kind suggest that stem cell transplantation, particularly using a donor’s cells, can offer meaningful long-term survival for patients who would otherwise face a grim prognosis.
HEPATOSPLENIC T-cell lymphoma is one of the rarest and most aggressive blood cancers, with no agreed standard treatment and a track record of poor responses to chemotherapy.
For this study, researchers retrospectively analysed outcomes for 121 adult patients with a confirmed or ‘consistent-with’ diagnosis of hepatosplenic T-cell lymphoma, treated across 64 centres in Europe and Asia
Of the cohort, 94 underwent allogeneic haematopoietic stem cell transplantation (allo-HSCT), using stem cells donated by another person, and the other 27 received an autologous transplant (auto-HSCT) using their own previously collected stem cells.
The analysis found that three years after an allogeneic transplant, just over half of patients (55%) were still alive and more than half (50.5%) had not relapsed or progressed.
It also showed that patients who entered transplant already in complete remission had close to three times better odds of survival than those who still had active disease.
Around a third of patients who went into transplant with active, progressive disease still achieved long-term survival, results comparable to no other available treatment option for hepatosplenic T-cell lymphoma.
Researchers stress that for this reason, patients with poorer prognostic markers should not automatically be ruled out of a donor transplant.
Additionally, patients with normal levels of lactate dehydrogenase (LDH), a simple and widely available blood test, at the time of diagnosis fared significantly better than those with raised levels.
Relapse remained the biggest threat to long-term success of the transplant, affecting around 38% of patients within three years. Death directly linked to the transplant procedure itself was relatively uncommon, at under 12%, something researchers attribute in part to the relatively young age of most patients, who were a median age of 36.
The team argue that these results point to a genuine, if partial, chance of cure, driven largely by the donor immune cells actively fighting residual lymphoma, an effect known as graft-versus-lymphoma.
Outcomes were markedly less encouraging for the smaller group of patients who received an autologous transplant.
Although representing a highly selected, lower-risk group, with the majority of these patients (74%) in complete remission before treatment, only around 39% remained progression-free at three years. Half of them had relapsed.
Due to this transplant approach relying predominantly on high-dose chemotherapy rather than a donor immune response to destroy remaining cancer cells, researchers suggest hepatosplenic T-cell lymphoma may simply be too resistant to chemotherapy for the benefit to last, even in patients who initially responded well to treatment.
With current treatment guidelines for hepatosplenic T-cell lymphoma resting on limited evidence from small case series, the authors say this large-scale analysis, more than double the size of any previous study, offers the most reliable outlook yet for patients and clinicians navigating this disease.
They recommend that eligible patients receive aggressive initial chemotherapy followed by rapid allogeneic transplantation wherever possible, reserving autologous transplant only for those who achieve complete remission but are not suitable candidates for a donor transplant.
The authors also acknowledged the study’s limitations in that it could only capture patients who went on to receive a transplant, offering no insight into those who were considered but never underwent the procedure.
Validating these findings in an even larger cohort is a logical next step, though the rarity of the disease will always constrain sample sizes.
Reference:Karsten I et al. Haematopoietic stem cell transplantation in hepatosplenic T-cell lymphoma: a retrospective analysis of the EBMT Lymphoma Working Party. The Lancet Haematology. 2026
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