Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years

We investigated the outcomes of 179 pediatric patients with severe aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (SAA) who received up-front hematopoietic stem cell transplantation (HSCT; n = 87) or received HSCT after failure of immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… (IST; n = 92). With a median follow-up of 4.0 years (range, 0.2-10.7) after HSCT, 161 patients were alive at last follow-up. The cumulative incidence of graft failure, grade 2 to 4 acute and chronic graft-versus-host disease (GVHD) graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… was 10%, 18%, and 8%, respectively. Five-year overall survival (OS) and GVHD-free, relapse/rejection-free survival (GRFS) were 89% and 79%, respectively. Patients who received up-front HSCT from a matched sibling donor (MSD) or ≥9/10 HLA HLA: See human leukocyte antigen. -matched unrelated donor unrelated donor: A donor that is not a sibling or other familial relation of the patient (recipient). (MUD) both achieved an OS of 95%. OS was superior in patients who received up-front HSCT than those who received HSCT after failure of IST (up-front, 95% [95% CI, 89-100] vs after IST failure, 83% [95% CI,75-91]; P < .01). However, OS in the HSCT group after IST failure improved over time, resulting in comparable OS between up-front HSCT and HSCT after IST failure from 2015 (97% vs 89%; P = .09). Moreover, there was no difference in GRFS of MUD-HSCT comparing patients who received a transplant up front or after IST failure (up-front, 77% vs after IST failure, 76%; P value not significant). Our data highlight the good transplant outcomes of a consecutive pediatric SAA cohort. Although up-front HSCT from a readily available MUD is a viable option if an MSD is not available, HSCT after IST failure from a well-matched unrelated donor also yields favorable outcomes.