<body><div>The majority of pediatric patients with Fanconi anemia (FA) who underwent hematopoietic stem cell transplantation (HSCT) were alive and event-free at 5 years, according to the results of a retrospective study published in the journal Blood.Researchers also found that the cumulative incidences (CINs) of primary and secondary graft failure and secondary malignancy were low.In the study, researchers analyzed data from 813 pediatric patients who underwent their first HSCT between 2010 and 2018. The cohort included 96% of patients with FA-associated bone marrow failure (BMF)/aplastic anemia (AA) and 4% with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The median follow-up was 3.7 years.The overall survival (OS) was 87% at 1 year and 83% at 5 years. The event-free survival (EFS) was 82% and 78% at 1 and 5 years, respectively. The 5-year graft vs host disease (GVHD)-free relapse-free survival (GRFS) was 70%.OS was similar among patients with matched family (MFD) or matched unrelated (MUD) doners at 88% and 86%, respectively. These rates were significantly higher than the 5-year OS among patients with a mismatched family (MMFD) donor or mismatched unrelated doner (MMUD) at 72% or haploidentical donors (HID) at 70% (P &lt;.001).In a multivariate analysis, the researchers found that shorter OS was significantly associated with AML or MDS (hazard ratio [HR], 4.47; 95% CI, 2.58-7.74; P &lt;.001), a MMFD/MMUD (HR, 2.03; 95% CI, 1.26-3.26; P =.004) or HID doner (HR, 2.48; 95% CI, 1.14-5.40; P =.02), and conditioning with FluCy plus other agents (HR, 1.81; 95% CI, 1.00-3.31; P =.05). Longer OS was associated with the use of alemtuzumab (HR, 0.33; 95% CI, 0.14-0.77; P =.01).<blockquote>The largest study ever conducted on HSCT in FA confirms excellent survival…longer follow-up is required to fully assess the risk of second cancer in this young study population.</blockquote>The most common causes of transplant-related mortality during the initial 5 years after transplantation were GVHD (33%) and infection (28%). The CIN of death at 5 years due to GVHD with or without therapy was 5% and 9%, respectively (P =.045), and was 5% and 2%, respectively, for infection (P =.2).The CIN of primary and secondary graft failure was 2% and 3%, respectively. The CIN if acute GVHD was 23% for grade 2-4 and 12% for grade 3-4 within the first 100 days after transplant. For chronic GVHD, the CIN was 8% at 1 year and 12% at 5 years. The 5-year CIN for a secondary malignancy was 2%. The most common secondary malignancies were solid tumors and leukemia.“In conclusion,” the researchers wrote, “the largest study ever conducted on HSCT in FA confirms excellent survival.” They added that “longer follow-up is required to fully assess the risk of second cancer in this young study population.”</div></body>

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HSCT Is Effective in Pediatric Patients With Fanconi Anemia

The majority of pediatric patients with Fanconi anemia (FA) who underwent hematopoietic stem cell transplantation (HSCT) were alive and event-free at 5 years, according to the results of a retrospective study published in the journal Blood. Researchers also found that the cumulative incidences (CINs) of primary and secondary graft failure...