Low toxicity and favorable clinical and quality of life impact after non-myeloablative autologous hematopoietic stem cell transplant in Crohn’s disease

Abstract

ObjectiveThe incidence of adverse events in myeloablative transplant protocols is high in refractory Crohn’s disease; this study used low doses of cyclophosphamide. Fourteen patients were submitted to non-myeloablative autologous hematopoietic stem cell transplantation.ResultsThe average number of days of anemia (hemoglobin < 10 g/dL) was 5.4 ± 4.2 and 14 ± 2.4 in the mobilization and conditioning phases, respectively. The mean number of days of neutropenia (neutrophils < 0.5 × 109/L) in the mobilization phase was 1.7 ± 1.5 while it was 7.6 ± 1.4 in the conditioning phase. When comparing the conditioning and mobilization phases, there was an increased number days of leukopenia (white blood cells < 1.0 × 109/L), lymphocytopenia (lymphocytes < 0.5 × 109/L) and thrombocytopenia (platelets < 25 × 109/L). Crohn’s Disease Activity Index values before the transplant ranged from 155 to 450.5 (mean 281.2 ± 79.0) and at 30 days after the procedures they ranged from 45.4 to 177 (mean 95.8 ± 35.4). Moreover, the procedure improved in overall quality of life of patients. Non-myeloablative autologous hematopoietic stem cell transplantation with lower doses of cyclophosphamide leads to lower rates of hematological toxicity and adverse events compared to protocols described in the literature. Trial registration NCT 03000296: Date 9 December 2016Electronic supplementary materialThe online version of this article (doi:10.1186/s13104-017-2824-1) contains supplementary material, which is available to authorized users.

Crohn’s disease patients effectively mobilize peripheral blood stem cells to perform autologous haematopoietic stem cell transplantation

“…The data are from the project “Autologous Unselected Haematopoietic Stem Cell Transplantation for Crohn’s Disease”, registered in the US platform Clinicaltrials.gov (NCT 03000296). Partial data of this series have been published previously (17).…”

Immune Reconstitution After Autologous Hematopoietic Stem Cell Transplantation in Crohn’s Disease: Current Status and Future Directions. A Review on Behalf of the EBMT Autoimmune Diseases Working Party and the Autologous Stem Cell Transplantation In Refractory CD—Low Intensity Therapy Evaluation Study Investigators

“…Subsequent analysis of the 38 patients who underwent auto-HSCT in the ASTIC program and had data at baseline and 1 year reported a significant reduction in clinical and endoscopic disease activity at 1 year, with 19 out of 38 (50%) patients showing regression of all endoscopic ulceration ( 17 ). There were also significant improvements in quality of life between baseline and 1 year after auto-HSCT ( 17 , 19 , 20 ). Importantly, disease recurrence after HSCT responded to the introduction of anti-TNF therapies ( 15 , 17 ).…”

“…The median duration of anemia and neutropenia was shorter after both mobilization and conditioning than that seen in previous reports using higher cyclophosphamide dosing, and few episodes of febrile neutropenia were reported. The lower intensity regimen still resulted in marked reduction in clinical disease activity with 13 patients achieving disease remission (CDAI < 150) at 30 days ( 20 ).…”

Revisión literaria sobre el trasplante de células madre hematopoyéticas, opciones para mejorar la asimilación y su impacto en la supervivencia de los pacientes

“…Algo que no es muy común es el uso del trasplante de células hematopoyéticas en la enfermedad de Crohn; es un tratamiento no convencional el cual tiene más riesgos que beneficios, incluyendo la reducción en el funcionamiento del órgano y susceptibilidad a infecciones. A pesar de que en un estudio menciona la mieloablación con falla irreversible de la médula ósea en la fase de acondicionamiento, posiblemente por las altas dosis de ciclofosfamida, se están explorando alternativas no mieloablativas [54].…”