Transplantation and cellular therapy · 2026
Infused CD34+ cell dose predicts response to stem cell boost in children with poor graft function following allogeneic hematopoietic stem cell transplantation: a multicenter study from the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).
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Benítez-Carabante María Isabel, Ortiz-Palacios Marina, Carbonell-Asins Juan Antonio, Enríquez Sara Vinagre, Uría-Oficialdegui María Luz, Mozo Del Castillo Yasmina, Torija Iván López, Sisinni Luisa, Molina Blanca, Fornieles Mercedes Plaza, Urtasun-Erburu Andrea, Socorro Carolina Fuentes, Culebras Marta García, Trabazo Del Castillo María, Sojo Jesús Fernández, Castillo-Flores Nerea, García Laura Alonso, González-Vicent Marta, Díaz-de-Heredia Cristina
Аннотация
BACKGROUND: Poor graft function (PGF) is a severe and potentially life-threatening complication after allogeneic hematopoietic stem cell transplantation (HSCT), characterized by persistent cytopenias despite adequate donor chimerism. Although CD34+ selected stem cell boost (CD34+SCB) has emerged as a promising treatment, evidence in pediatric patients remains limited, and the optimal CD34+ cell dose required to achieve complete hematologic recovery has not been established.
OBJECTIVES: To evaluate outcomes of CD34+SCB in children with PGF, identify factors associated with hematologic recovery and survival, and determine the CD34+ cell dose required to achieve complete hematologic recovery with external validation.
METHODS: We conducted a national multicenter retrospective study. Pediatric patients (≤18 years) with severe PGF involving at least two hematopoietic lineages who received CD34+SCB from the original donor were included in the training cohort (n=42). Primary endpoint was complete response (CR). Secondary endpoints included overall response rate (ORR), overall survival (OS) and factors associated with CR and OS. The optimal CD34+ cell dose threshold predicting CR was identified using maximally selected rank statistics and externally validated with an independent cohort.
RESULTS: Median time from HSCT to PGF diagnosis was 89 days, and 88% of patients presented with trilineage cytopenia. The median infused CD34+ cell dose was 7.3 × 106/kg. ORR and CR were 83.3% and 61.9%, respectively, with median time to CR of 43 days. With a median follow-up of 19.9 months, 2-year OS was 59.5%. Most deaths were due to infections and occurred in non-responders. In multivariable analysis, infused CD34+ cell dose was the only independent factor predictor of CR (HR 1.14, 95% CI 1.02-1.26; p=0.019). A threshold of 4.65 × 10⁶ CD34+ cells/kg significantly discriminated the probability of achieving CR in the training cohort (p=0.016) and was independently validated in the external cohort (p=0.013). Previous chronic graft-versus-host disease (GVHD) and haploidentical transplantation were independently associated with inferior OS. CD34+SCB demonstrated a favorable safety profile, with low incidences of de novo acute and chronic GVHD CONCLUSION: CD34+SCB is an effective and safe therapeutic strategy for pediatric PGF following HSCT, achieving high hematologic recovery rates with acceptable long-term survival. Infused CD34+ cell dose is the main determinant of complete hematologic recovery, and a minimum threshold may help optimize treatment outcomes. Further studies are warranted to refine risk-adapted approaches in this high-risk population.
OBJECTIVES: To evaluate outcomes of CD34+SCB in children with PGF, identify factors associated with hematologic recovery and survival, and determine the CD34+ cell dose required to achieve complete hematologic recovery with external validation.
METHODS: We conducted a national multicenter retrospective study. Pediatric patients (≤18 years) with severe PGF involving at least two hematopoietic lineages who received CD34+SCB from the original donor were included in the training cohort (n=42). Primary endpoint was complete response (CR). Secondary endpoints included overall response rate (ORR), overall survival (OS) and factors associated with CR and OS. The optimal CD34+ cell dose threshold predicting CR was identified using maximally selected rank statistics and externally validated with an independent cohort.
RESULTS: Median time from HSCT to PGF diagnosis was 89 days, and 88% of patients presented with trilineage cytopenia. The median infused CD34+ cell dose was 7.3 × 106/kg. ORR and CR were 83.3% and 61.9%, respectively, with median time to CR of 43 days. With a median follow-up of 19.9 months, 2-year OS was 59.5%. Most deaths were due to infections and occurred in non-responders. In multivariable analysis, infused CD34+ cell dose was the only independent factor predictor of CR (HR 1.14, 95% CI 1.02-1.26; p=0.019). A threshold of 4.65 × 10⁶ CD34+ cells/kg significantly discriminated the probability of achieving CR in the training cohort (p=0.016) and was independently validated in the external cohort (p=0.013). Previous chronic graft-versus-host disease (GVHD) and haploidentical transplantation were independently associated with inferior OS. CD34+SCB demonstrated a favorable safety profile, with low incidences of de novo acute and chronic GVHD CONCLUSION: CD34+SCB is an effective and safe therapeutic strategy for pediatric PGF following HSCT, achieving high hematologic recovery rates with acceptable long-term survival. Infused CD34+ cell dose is the main determinant of complete hematologic recovery, and a minimum threshold may help optimize treatment outcomes. Further studies are warranted to refine risk-adapted approaches in this high-risk population.