Abstract
Background. The pediatric hematopoietic stem cell transplantation (HSCT) program at VUHSK
was launched in 2002. It provides autologous and allogeneic services for all Lithuanian and, since
2011, Latvian children.
Aims. We aimed to assess change in HSCT outcomes over two decades.
Methods. A retrospective analysis of all consecutive HSCTs from 2002 to 2021 was performed.
Two time periods (2002-2011 and 2012-2021) were compared to evaluate the change. The 5-year
overall survival (OS5y), the cumulative incidence of relapse (CIR) in the malignant setting and transplant-related mortality (TRM) were analyzed. Descriptive statistics and Kaplan-Meier survival estimates were calculated.
Results. From 2002 to 2021 totally 269 HSCTs (56.5% (152/269) allogeneic and 43.5% (117/269)
autologous) were performed in 246 unique recipients. The median annual number of allogeneic
HSCT increased from 6 in 2002-2011 to 11 in 2012-2021. Non-Lithuanian citizens comprised 22.2%
of all unique recipients (32/144) in 2012-2021.
In the autologous setting, no changes occurred comparing OS5y, CIR and TRM in 2002-2011
(n=52/103) vs 2012-2021 (n=51/103).
In the allogeneic setting a significant improvement in OS5y from 0.380 (95% CI 0.267, 0.541) in
2002-2011 (n=50/144) to 0.768 (95% CI 0.686, 0.861) in 2012-2021 (n=93/144), p < 0.0001, was
observed. The TRM at 100 days decreased from 0.260 (95% CI 0.143, 0.380) in 2002-2011 to 0.099
(95% CI 0.046, 0.172) in 2012-2021, p <0.0001 and at 5 years – from 0.507 (95% CI 0.353, 0.643)
to 0.165 (95% CI 0.097, 0.249), p < 0.0001, respectively. None of the patients transplanted from an
HLA-identical sibling in 2012-2021 (n=25) succumbed due to TRM.
The subgroup analysis of 85 patients transplanted for malignant diseases showed a significant
decrease in the CIR5y from 0.435 (95% CI 0.213, 0.639) in 2002-2011 (n=34/85) to 0.156 (95% CI
0.062, 0.290) in 2012-2021 (n=51/85), p = 0.043.
Conclusions. Despite a small transplant volume, a significant improvement over two decades
was demonstrated. A close collaboration between Lithuania and Latvia and centralized patient referral is essential to maintain sufficient transplant volume and ensure safe transplant quality
was launched in 2002. It provides autologous and allogeneic services for all Lithuanian and, since
2011, Latvian children.
Aims. We aimed to assess change in HSCT outcomes over two decades.
Methods. A retrospective analysis of all consecutive HSCTs from 2002 to 2021 was performed.
Two time periods (2002-2011 and 2012-2021) were compared to evaluate the change. The 5-year
overall survival (OS5y), the cumulative incidence of relapse (CIR) in the malignant setting and transplant-related mortality (TRM) were analyzed. Descriptive statistics and Kaplan-Meier survival estimates were calculated.
Results. From 2002 to 2021 totally 269 HSCTs (56.5% (152/269) allogeneic and 43.5% (117/269)
autologous) were performed in 246 unique recipients. The median annual number of allogeneic
HSCT increased from 6 in 2002-2011 to 11 in 2012-2021. Non-Lithuanian citizens comprised 22.2%
of all unique recipients (32/144) in 2012-2021.
In the autologous setting, no changes occurred comparing OS5y, CIR and TRM in 2002-2011
(n=52/103) vs 2012-2021 (n=51/103).
In the allogeneic setting a significant improvement in OS5y from 0.380 (95% CI 0.267, 0.541) in
2002-2011 (n=50/144) to 0.768 (95% CI 0.686, 0.861) in 2012-2021 (n=93/144), p < 0.0001, was
observed. The TRM at 100 days decreased from 0.260 (95% CI 0.143, 0.380) in 2002-2011 to 0.099
(95% CI 0.046, 0.172) in 2012-2021, p <0.0001 and at 5 years – from 0.507 (95% CI 0.353, 0.643)
to 0.165 (95% CI 0.097, 0.249), p < 0.0001, respectively. None of the patients transplanted from an
HLA-identical sibling in 2012-2021 (n=25) succumbed due to TRM.
The subgroup analysis of 85 patients transplanted for malignant diseases showed a significant
decrease in the CIR5y from 0.435 (95% CI 0.213, 0.639) in 2002-2011 (n=34/85) to 0.156 (95% CI
0.062, 0.290) in 2012-2021 (n=51/85), p = 0.043.
Conclusions. Despite a small transplant volume, a significant improvement over two decades
was demonstrated. A close collaboration between Lithuania and Latvia and centralized patient referral is essential to maintain sufficient transplant volume and ensure safe transplant quality
| Original language | English |
|---|---|
| Pages (from-to) | 366 |
| Journal | Acta Medica Lituanica. |
| Volume | 29 |
| Issue number | 2/Suppl. |
| Publication status | Published - 2022 |
| Event | Pediatric hematopoietic stem cell transplantation in Lithuania – 20 years of Progress through collaboration - Radisson Blu, Vilnius, Lithuania Duration: 22 Sept 2022 → 23 Sept 2022 https://www.santa.lt/uploads/Programa_vaikų%20KKLT%2020%20mečiui_eng.pdf |
Field of Science*
- 3.2 Clinical medicine
- 3.3 Health sciences
Publication Type*
- 3.4. Other publications in conference proceedings (including local)