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Allogeneic hematopoietic cell transplantation (allo-HCT) remains a curative option for patients with high-risk myeloid malignancies.We present our 10-year experience (October 2012 to October 2021) of consecutive allo-HCT in patients with myeloid malignancies treated on the pediatric HCT service and conditioned with myeloablative targeted dose-busulfan (BU), fludarabine (FLU), and melphalan (MEL). Twenty-three children, adolescents, and young adult patients (CAYA) (median age 15.4 years) with acute myeloid leukemia (AML, n = 17), myelodysplastic syndrome (MDS, n = 4), or chronic myeloid leukemia (CML, n = 2) underwent allo-HCT post-BU-FLU-MEL. Four patients had treatment-related AML/MDS. Donor/stem cell source was matched sibling donor (MSD) PBSC (n = 7), matched unrelated donor (MUD) PBSC (n = 2), umbilical cord blood (UCB) (n = 3), or haploidentical-BMT (n = 11). Risk stratification was low (n = 2), intermediate (n = 15), high (n = 3), and very high risk (n = 1). The two patients with CML had failed tyrosine kinase inhibitor therapies.With a median follow-up of 41.6 months, the relapse rate is only 4.5% with an overall survival (OS) 100%, progression-free survival (PFS) 95.5%, and graft-versus-host-free-relapse-free survival (GRFS) 67.8%. The donor source and the acute graft-versus-host disease (GvHD) prophylaxis regimen significantly impacted grade II-IV aGvHD 66.7% versus 19.2% (p = .039) and chronic graft-versus-host-disease (cGvHD) 66.7% versus 0% (p = .002) in the patients receiving MSD or MUD PBSC compared to haplo-BMT, respectively, resulting in improved GRFS in haplo-BMT, 83.3% compared to 40% matched donor peripheral blood stem cell transplant (PBSCT) (p = .025).Our results demonstrate that BU-FLU-MEL is efficacious conditioning for disease control in young patients with myeloid malignancies undergoing MSD or alternative donor allo-HCT, but in the setting of PBSC grafts with cyclosporine A-methotrexate (CSA-MTX) GvHD prophylaxis, it results in an unacceptably high incidence of GvHD.

Corticosteroids play an essential role in the treatment of pediatric malignancies, but have many untoward side effects including behavioral and mood disturbances which can be quite burdensome to families. Potassium chloride has been used anecdotally to decrease these neuropsychiatric effects but this experience has not been studied systematically. We therefore retrospectively reviewed our experience utilizing KCl supplementation to reduce corticosteroid-induced neuropsychiatric effects among children with acute lymphoblastic leukemia. Thirteen of 16 patients (81%) had a objective benefit with KCl at a median dose of 0.5 mEq/kg/day, with no reported adverse effects. Further prospective study is required to confirm these data.

Ovarian angiosarcoma is a rare and aggressive vascular tumor, which has a 5-year overall survival of less than 30% for patients with nonmetastatic disease and almost certain death within 1 year for those with metastasis. Here, we briefly review historical approaches to therapy and present a long-term survivor in the case of an 11-year-old female with metastatic ovarian angiosarcoma. This is the second reported case to utilize heated intraperitoneal chemotherapy in the treatment of this disease. Our patient is currently alive and well 3 years after initial diagnosis, significantly longer than any reported case of advanced-stage ovarian angiosarcoma.