Unmanipulated Haploidentical Hematopoietic Stem Cell Transplantation Achieved Outcomes Comparable With Matched Unrelated Donor Transplantation in Young Acquired Severe Aplastic Anemia

Abstract

Salvage haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is considered in patients with severe aplastic anemia (SAA) if a matched unrelated donor (MUD) is unavailable. However, studies on haplo- and MUD transplantation in SAA are lacking. The present study retrospectively analyzed the outcomes of 89 young SAA patients who underwent unmanipulated alternative HSCT between September 2012 and September 2016 at our single center. Forty-one patients received haploidentical donors and forty-eight patients MUDs for HSCT. Most were heavily transfused and refractory to previous immunotherapy. The median durations for myeloid engraftment in the haplo- and MUD cohorts were 14 (range, 10 to 21) and 13 (range, 10 to 18) days, respectively. Compared with the MUD cohort, haplo-HSCT cohorts had an increased cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV (43.9% ± 7.8% versus 12.5% ± 4.8%, P = .001) and grades III to IV (21.1% ± 6.7% versus 6.6% ± 3.7%, P = .045) and similar limited chronic GVHD (47.7% ± 8.5% versus 38.5% ± 7.3%, P = .129) and extensive chronic GVHD (12.1% ± 6.8% versus 9.1% ± 4.3%, P = .198). The median follow-up time of the surviving patients was 26 months (range, 6 to 45). No significant differences were observed between haplo-HSCT and MUD HSCT cohorts in 3-year overall survival (80.3% ± 5.1% versus 89.6% ± 7.0%, P = .210), disease-free survival (76.4% ± 5.1% versus 89.4% ± 7.7%, P = .127), and GVHD-free failure-free survival (79.0% ± 8.6% versus 71.6% ± 9.3%, P = .976). Thus, haplo-HSCT, as salvage therapy, achieved similar outcomes as MUD HSCT in young SAA patients, thereby rendering it as an effective and safe option for SAA.

Comparable survival outcome between transplantation from haploidentical donor and matched related donor or unrelated donor for severe aplastic anemia patients aged 40 years and older: A retrospective multicenter cohort study

“…With the advantages of easy donor availability and short preparation time for transplantation, HID‐HSCT was regarded as a potential and promising therapy for patients without MSD and URD. Moreover, HID‐HSCT had achieved remarkable progress and showed comparable outcomes with HSCT from matched donors in younger SAA patients . Nevertheless, the outcome of HSCT from HID in SAA patients 40 years of age and older remains unknown.…”

“…Moreover, HID-HSCT had achieved remarkable progress and showed comparable outcomes with HSCT from matched donors in younger SAA patients. [8][9][10][11][12] Nevertheless, the outcome of HSCT from HID in SAA patients 40 years of age and older remains unknown.…”

Desenlace de los pacientes pediátricos con falla medular tratados en un centro de alta complejidad

“…Sin embargo, también pueden emplearse de sangre periférica o proveniente de sangre de cordón umbilical 32 . Estudios recientes muestran buenos resultados utilizando trasplantes haploidénticos como primera línea o de rescate, incluso resultados comparables con donante relacionado idéntico, lo cual los convierte en una alternativa segura [33][34][35] . En esta serie se reportan igual número de TPH con donante idéntico y haploidéntico, con una SG del 84%, lo cual coincide con reportes recientes en donde las tasas de SG son similares con diferentes tipos de donante 13,26,33,34,36 .…”

“…Estudios recientes muestran buenos resultados utilizando trasplantes haploidénticos como primera línea o de rescate, incluso resultados comparables con donante relacionado idéntico, lo cual los convierte en una alternativa segura [33][34][35] . En esta serie se reportan igual número de TPH con donante idéntico y haploidéntico, con una SG del 84%, lo cual coincide con reportes recientes en donde las tasas de SG son similares con diferentes tipos de donante 13,26,33,34,36 . En esta serie se observó EICH agudo y crónico en el 29% de los pacientes, similar a lo reportado en la literatura con diferentes tipos de donante, que están entre 21,1-47,7% 26,34,37 .…”

“…En esta serie se reportan igual número de TPH con donante idéntico y haploidéntico, con una SG del 84%, lo cual coincide con reportes recientes en donde las tasas de SG son similares con diferentes tipos de donante 13,26,33,34,36 . En esta serie se observó EICH agudo y crónico en el 29% de los pacientes, similar a lo reportado en la literatura con diferentes tipos de donante, que están entre 21,1-47,7% 26,34,37 . El estudio realizado por Sallfors-Holmqvist et al (BMTSS-2), entre 1974 y 2010, en el cual incluyeron pacientes menores de 21 años llevados a TPH debido a SFM, obtuvieron una SG de 86,4% a los 15 años posterior al TPH 38 .…”