Handling missing covariate data in clinical studies in haematology.

Long term survivor of MOZ-p300 therapy-related acute myeloid leukemia after matched unrelated donor bone marrow transplantation.

Allogeneic hematopoietic cell transplantation (allo‐HCT) remains a curative option for patients with high‐risk myeloid malignancies.

Busulfan, fludarabine, and melphalan are effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

Abstract Background: Allogeneic hematopoietic cell transplantation (allo-HCT) remains a curative option for patients with high-risk myeloid malignancies. Objectives : We reviewed our ten-year experience (October 2012 thru October 2021) of 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). Study Design: Twenty-three children, adolescents, and young adult patients (CAYA) (median age 15.4 years, range 0.6 to 27.2) with acute myeloid leukemia (AML) (n=17), myelodysplastic syndrome (MDS) (n=4), or chronic myeloid leukemia (CML) (n=2) underwent allo-HCT conditioned with BU-FLU-MEL. Four patients had treatment-related AML/MDS, following osteogenic and Ewing sarcoma (t-AML, n=2) and acute lymphoblastic leukemia (t-MDS, n=2). Donor/stem cell source was matched sibling donor (MSD) peripheral blood stem cells (PBSC) (n=7), matched unrelated donor (MUD) PBSC (n=2), unrelated single (n=1) or double umbilical cord blood (UCB) (n=2) or haploidentical donor bone marrow (haplo-BMT) (n=11). Graft-versus-host disease (GvHD) prophylaxis consisted of cyclosporine-methotrexate (CSA-MTX) for MSD and MUD, and CSA-mycophenolate mofetil (MMF) for unrelated UCB transplants. Patients undergoing T-cell replete haplo-BMT received post-transplant cyclophosphamide (PT-CY) (n=7) or cyclophosphamide and bendamustine PT-CY/BEN (n=4) with tacrolimus and MMF. Thirteen patients with of AML were in first remission (CR1) with seven being minimal residual disease negative (MRD - ), three patients in second remission (CR2) with two MRD - , and five patients had received no treatment prior to allo-HCT (MDS, n=4 and AML evolved from del(7q) MDS). Risk stratification based on remission/MRD status, cytogenetics and age was low (n=2), intermediate (n=15), high (=3) and very high risk (n=1). The two patients with CML had failed tyrosine kinase inhibitor therapies. Results: With a median follow-up of 37.2 months the relapse rate is only 4.5% with a remarkable overall survival (OS) of 100%, progression-free survival (PFS) of 95.5% and graft-versus-host-free relapse-free survival (GRFS) of 67.8%. The donor source and the graft-versus-host-disease (GvHD) prophylaxis regimen significantly impacted the cumulative incidence of grades II-IV acute GvHD (aGvHD) 66.7% versus 18.2% ( P=0.03 ) and chronic GvHD (cGvHD) 66.7% versus 0% ( P=0.001 ) in the patients receiving MSD or MUD PBSC compared to haplo-BMT respectively. This resulted in significantly improved GRFS in haplo-BMT recipients of 83.3% compared to 40% in patients receiving related or unrelated matched donor PBSC transplantation ( P=0.025 ). Conclusions: Our results demonstrate that BU-FLU-MEL is an efficacious conditioning regimen for disease control in young patients with myeloid malignancies undergoing matched sibling or alternative donor hematopoietic cell transplantation but in the setting of PBSC grafts with CSA-MTX GvHD prophylaxis it results in an unacceptably high incidence of GvHD. 

Busulfan Fludarabine and Melphalan is effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

Myeloid Neoplasms Postcytotoxic Therapy in Children

Long-term outcomes after allogeneic hematopoietic cell transplantation (HCT) for therapy-related myeloid neoplasms (tMNs) are dismal. There are few multicenter studies defining prognostic factors in pediatric patients with tMNs. We have accumulated the largest cohort of pediatric patients who have undergone HCT for a tMN to perform a multivariate analysis defining factors predictive of long-term survival. Sixty-eight percent of the 401 patients underwent HCT using a myeloablative conditioning (MAC) regimen, but there were no statistically significant differences in the overall survival (OS), event-free survival (EFS), or cumulative incidence of relapse and non-relapse mortality based on the conditioning intensity. Among the recipients of MAC regimens, 38.4% of deaths were from treatment-related causes, especially acute graft versus host disease (GVHD) and end-organ failure, as compared to only 20.9% of deaths in the reduced-intensity conditioning (RIC) cohort. Exposure to total body irradiation (TBI) during conditioning and experiencing grade III/IV acute GVHD was associated with worse OS. In addition, a diagnosis of therapy-related myelodysplastic syndrome and having a structurally complex karyotype at tMN diagnosis were associated with worse EFS. Reduced-toxicity (but not reduced-intensity) regimens might help to decrease relapse while limiting mortality associated with TBI-based HCT conditioning in pediatric patients with tMNs.

Outcomes of pediatric patients with therapy-related myeloid neoplasms.

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Outcomes of pediatric patients with therapy-related myeloid neoplasms.