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Background: There is paucity of data regarding outcomes of children, adolescents and young adults (CAYA) patients with non-Hodgkin lymphoma (NHL) undergoing autologous stem cell transplantation (ASCT)., Methods: Patients aged 0-39 years undergoing first ASCT for NHL at MD Anderson Cancer Center between 2000 and 2020 were analyzed., Results: Two hundred twenty-one patients were included in the analysis, 129 (58%) were male and the median age was 32 (range 6-39) years. The most common histological subtypes were diffuse large B cell lymphoma (DLBCL) (44%), T-NHL (19%) and primary mediastinal B-Cell lymphoma (PMBCL) (19%). Younger patients (age ≤ 25) had lower incidence of DLBCL and higher incidence of PMBCL and T-NHL compared to older patients (age > 25) (P = 0.02). None of the younger patients had double hit (DH)/double expressor (DE) DLBCL, compared to 14 patients in the older age group (18%, P = 0.07). Considering the three main aggressive NHL subtypes (DLBCL, PMBCL and T-NHL), younger patients had numerically better 15-year post-transplant progression free survival (PFS) (67% vs. 54%) and overall survival (OS) (71% vs. 62%) compared to older patients, yet these differences did not reach statistical significance (P = 0.19 and P = 0.24, respectively). In multivariate analysis, not achieving a CR prior to ASCT was independently predictive of worse PFS [partial remission (PR) (HR, 3.9); stable disease (SD) (HR, 18.0), P = 0.03] and of worse OS [PR (HR, 4.2), SD (HR, 6.5) and progressive disease (HR, 4.7), P < 0.0001]. DH/DE status was an independent adverse predictor of PFS in multivariate analysis (HR 5.8, p = 0.03). Ten patients (4.5%) (all aged > 25 years) developed second primary malignancies (SPM), at a median of 34.4 (range, 1.0-196.6) months after ASCT, and SPM was the cause of death in five (50%) of them., Conclusions: CAYA NHL patients aged ≤ 25 years who received ASCT presented a distinct NHL histology as compared to older CAYA patients, and none in this younger age group had DH/DE DLBCL. We observed a trend towards improved PFS and OS in younger patients. Disease status at ASCT was predictive of both PFS and OS. DH/DE status was an adverse predictor of PFS., (© 2023. The Author(s).)

Summary: Multiple myeloma (MM) primarily affects older patients. There are scarce data on the outcomes of young adults undergoing autologous transplantation (auto‐HCT). In this single‐centre analysis, we included 117 younger patients, with a median age of 37 years (range 22–40) at transplant. Seventeen (15%) patients had high‐risk cytogenetics. Before transplant, 10% of patients achieved ≥CR and 44% achieved ≥VGPR. At best post‐transplant response, 56% and 77% of patients achieved ≥CR and ≥VGPR respectively. With a median follow‐up for survivors of 72.6 months (range 0.9–238.0), median PFS and OS were 43.1 months (95% CI 31.2–65.0) and 146.6 months (95% CI 100.0–208.1) respectively. Patients who underwent auto‐HCT after 2010 had better median PFS (84.9 months vs. 28.2 months, p < 0.001) and OS (NR vs. 91.8 months, p < 0.001) compared with those transplanted earlier. In multi‐variate analysis, achieving ≥CR as best post‐transplant response was associated with improved PFS (HR [95% CI] 0.55 [0.32–0.95], p = 0.032), while achieving ≥VGPR was predictive of superior OS (0.32 [0.16–0.62], p < 0.001). Three patients (3%) developed a second primary malignancy. Younger MM patients had durable survival after auto‐HCT, which further improved after the availability of novel anti‐myeloma drugs in recent years. Depth of response following transplant remains a key predictor of survival. [ABSTRACT FROM AUTHOR]

We conducted a retrospective analysis of outcomes for children and young adults with sAML/ sMDS who underwent HSCT at our institution. Thirty-two patients (median age 20 years) with sAML (n=24) and sMDS (n=8) received HSCT between 1990 and 2013. The median time from sAML/ sMDS diagnosis to HSCT was 4.1 months (range: 1.2-27.2 months). The transplant regimens were primarily busulfan based (n=19). BM was the primary donor source (n=15). Eleven recipients were transplanted with residual disease. At a median follow-up of 62.3 months (range: 0.4-250.9 months), 14 patients had disease recurrence. Acute GVHD, grade III/ IV, occurred in three patients. Causes of death were as follows: disease relapse (n=12), infection (n=2), pneumonia (n=1), pulmonary hemorrhage (n=1), acute GVHD (n=1), and graft failure (n=1). A PS of ≥90% at the time of HSCT had a significant impact on PFS ( P=.02). Patients achieving pretransplant primary CR (n=8) and those with sMDS and RA (n=6) had prolonged PFS ( P=.04). On multivariate analysis, shorter time to transplantation (≤6 months from diagnosis of sAML/ sMDS) was associated with superior OS ( P=.0018) and PFS ( P=.0005). [ABSTRACT FROM AUTHOR]

Background: Adolescents and young adults (AYAs) are a very unique subset of our population journeying through a dynamic stage of their lives. This age group often remains understudied as a separate entity because they are commonly lumped into either pediatric or adult subgroups. Methods: Here we review acute and chronic issues surrounding hematopoietic stem cell transplantation (HSCT) with a focus on the AYA age group. Results: HSCT is a commonly used treatment modality for patients with certain types of cancers. AYA patients undergoing HSCT present a very unique perspective, circumstances, medical, psychological and social issues requiring a diligent workup, care and follow-up. Conclusion: The medical care of these patients should be approached in a multidisciplinary method involving the patient, caregivers, physicians, psychologists and social workers. © 2014 S. Karger AG, Basel [ABSTRACT FROM AUTHOR]

Simple Summary: We present a case of a pediatric patient with symptomatic leukemia involving the heart without bone marrow involvement. The case is followed by a literature review of advances in leukemia treatment including immunotherapy, CAR T-cell therapy, improvement in the treatment of graft-versus-host disease, and the applicability of these treatments to leukemia involving areas outside of the bone marrow. We also explore the feasibility of treatments such as CAR T-cell therapy and blinatumomab and the associated risks for treating patients with leukemia infiltrating the heart. Isolated extramedullary relapse of acute lymphoblastic leukemia (ALL) occurs in soft tissues and various organs outside the testis and central nervous system. Treatments such as hematopoietic stem cell transplantation and more novel modalities such as immunotherapy have eradicated ALL at extramedullary sites. In some instances, survival times for relapsed ALL at these sites are longer than those for relapsed disease involving only the bone marrow. Isolated relapse of ALL in the myocardium is rare, especially in children, making diagnosis and treatment of it difficult. More recent treatment options such as chimeric antigen receptor T-cell therapy carry a high risk of cytokine release syndrome and associated risk of worsening cardiac function. Herein we present the case of an 11-year-old boy who presented with relapsed symptomatic B-cell ALL in the myocardium following allogeneic hematopoietic stem cell transplantation. This is an unusual presentation of relapsed ALL and this case demonstrates the associated challenges in its diagnosis and treatment. The case report is followed by a literature review of the advances in treatment of pediatric leukemia and their application to extramedullary relapse of this disease in particular. [ABSTRACT FROM AUTHOR]

Previous studies in adults have shown that peripheral blood absolute lymphocyte and monocyte count ratio (ALC/AMC) after autologous stem cell transplantation (ASCT) can predict outcome in patients with relapsed/refractory (R/R) Hodgkin lymphoma (HL). We retrospectively reviewed all of our children, adolescent, and young adult (CAYA) patients (age ≤26) who underwent transplantation for R/R HL between 2004 and 2015. Seventy-six patients (median age, 21; range, 10 to 26 years) who reached day 100 disease free were analyzed; 33% of them had positron emission tomography (PET)–positive tumors before ASCT. Patients received high-dose carmustine, etoposide, cytarabine, and melphalan (n = 40) or gemcitabine/busulfan/melphalan (n = 36). Median follow-up after day 100 was 3.9 years (95% confidence interval [CI], 2.8 to 4.9). A day 100 ALC/AMC ratio >2.1 correlated with lower risk of relapse (hazard ratio,  .097; 95% CI, .03 to .29; P  <   .0001). Patients with day 100 ALC/AMC ratios >2.1 and ≤2.1 had 4-year relapse-free survival rates of 93% and 33%, respectively ( P  = .0001) and 4-year overall survival rates of 96% and 76%, respectively ( P  = .0001). In addition, an ALC/AMC ratio increase >1.8 from day 15 to day 100 correlated with lower risk of relapse (hazard ratio, .24; 95% CI, .08 to 0.73; P  = .01). Likewise, an ALC/AMC ratio change >.26 from day 30 to day 100 also correlated with a lower likelihood of relapse (hazard ratio, .20; 95% CI,  .081 to .51; P  = .0007). Multivariate analysis showed that a positive PET scan at ASCT, day 100 ALC/AMC ratio ≤ 2.1, and an ALC/AMC ratio change either ≤1.8 from day 15 to day 100 or ≤.26 from day 30 to day 100 were independent adverse predictors. In conclusion, our analysis confirms in CAYA patients prior observations in adults indicating a major prognostic effect of peripheral lymphocyte and monocyte counts at day 100 and earlier post-ASCT time points in R/R HL. [ABSTRACT FROM AUTHOR]

Patients undergoing chemotherapy and especially hematopoietic stem cell transplantation (HSCT) require frequent transfusions, and thus, transfusion reaction (TR) vigilance is critical in this population. The reported incidence of TRs across all patients is 2%, and pediatric patients may experience 2-2.6 times more reactions than adults. However, the TR profile may differ in the immunocompromised population. There is a paucity of data in this area, as pediatric oncology and HSCT patients are often excluded from TR studies. Our goals were to assess the prevalence and describe TRs among immunocompromised pediatric patients and to improve recognition of TRs across this population. Pediatric clinical providers completed a TR training module in May – June, 2019. A single-institution retrospective review was conducted. All TRs in patients aged less than 25 years were reviewed over a 3-month period from July 1, 2019 to October 1, 2019. Over a 3-month period, 1,786 transfusions were administered in our designated population. Of those, 50.4% were platelets, 44.0% were red blood cells (RBC), 3.4% were cryoprecipitate, 1.4% were thawed plasma, and 0.8% were granulocytes. Those undergoing HSCT consumed 27.7% of total transfusions. Of all transfusions, there were 48 (2.7%) reported as possible TRs by nursing. Of those, 30 (1.7% of all transfusions) were adjudicated as true TRs. Fourteen (46.7%) of these true TRs were in patients with leukemia, 9 (30.0%) with HSCT, 5 (16.7%) with solid tumors, and 2 (6.7%) with aplastic anemia. Of the 9 reactions in HSCT patients, 4 occurred in allogeneic transplant recipients (3 umbilical cord blood and one matched unrelated donor) and 5 were in those receiving autologous HSCT. In HSCT patients, 1.8% of transfusions resulted in TRs compared to 1.6% in non-HSCT patients. Of the products triggering reactions, platelets accounted for 22 TRs (73.3%) and RBCs accounted for 8 TRs (26.7%). Of all 30 TRs, 15 (50%) were classified as febrile non-hemolytic transfusion reactions (FNHTR), 14 (46.7%) were considered allergic reactions, and 1 (3.3%) was diagnosed as transfusion-associated circulatory overload. Pre-medication was given in 22 (76.7%) of transfusions causing reactions. Of the 18 reports that were not adjudicated as true TRs, most (n=15) were generated for fever, but deemed unrelated to transfusion. Our baseline analysis revealed a higher prevalence than our historical institutional prevalence (1.7% vs. 1.3%), consistent with higher rates of TRs in the pediatric population, but lower than the 2% quoted in the literature, suggesting either underreporting or a different TR phenotype in this population. This population had a higher prevalence of FNHTR than the general pediatric population (50.0% vs. 31.5%). High rates of pre-medication and comorbidities (resulting in fevers) may influence TR presentation in this population. [ABSTRACT FROM AUTHOR]