A Tri-Institutional Approach to Address Disparities in Children's Oncology Group Clinical Trial Accrual for Adolescents and Young Adults and Underrepresented Minorities.

Purpose: Enrollment in Children's Oncology Group (COG) clinical trials has led to significant improvements in survival; however, disparities in survival persist, particularly among ethnic minorities, adolescents and young adults (AYAs), and the underinsured, partly due to inadequate access to cooperative group cancer clinical trials. In 2008, two COG sites University of Illinois at Chicago (UIC) and Rush University Medical Center, and a nonmember institution, John H Stroger Hospital, created a unified COG program utilizing one lead Institutional Review Board and research team. This study assesses the impact that the tri-institutional COG program had on clinical trial accrual for minority, AYA, and uninsured patients. Methods: Analysis and comparison of COG enrollment data from 2002 to 2008 (pre-merger) and 2008 to 2017 (post-merger) by age, ethnicity, insurance type, clinical trial type, oncologic diagnosis, and specialty of the enrolling physician were completed. Results: Following the merger, the total studies open to enrollment increased by 100%, enrollments increased by 446%, and, for each diagnoses, increased by more than 200%. Enrollment of ethnic minorities rose by 533%, most significantly for Hispanic patients by 925%. AYA enrollments increased by 822%. There was a 28-fold increase in enrollment of uninsured patients. Significantly more providers from various oncology specialties were engaged in enrolling patients and a consistent increase in the percentile standing of the program occurred after the merger. Conclusions: Creation of a tri-institutional COG research program was associated with significant increases in clinical trial enrollments, especially for underrepresented minorities, AYAs, and uninsured patients. The UIC/Rush/Stroger COG Program provides a novel and exemplary approach to address cancer health disparities for these vulnerable populations.

Recurrent Severe Iron Deficiency Anemia and Thrombocytopenia in an Adolescent Male.

An adolescent male presented with recurrent episodes over several years of severe iron deficiency anemia and associated severe thrombocytopenia. The anemia was secondary to chronic blood loss due to ulceration at the site of an ileocolonic anastomosis performed during infancy. We were able to demonstrate complete resolution of thrombocytopenia with the administration of iron, and without using steroids, intravenous immunoglobulin, or platelet transfusions. This is the first reported case of an individual with multiple episodes over several years of thrombocytopenia secondary to recurrent severe iron deficiency anemia, illustrating a predisposition to this complication in a unique patient.

A Rare Case of Spontaneous Remission of Terminal Deoxynucleotidyl Transferase Negative B-acute Lymphoblastic Leukemia.

Spontaneous remission of untreated pediatric leukemia is an extremely rare occurrence. The underlying mechanism may be because of an immune-mediated process or increased cortisol production during stress or infection. We describe a rare case of terminal deoxynucleotidyl transferase negative B-acute lymphoblastic leukemia with concurrent infection that went into remission without treatment with chemotherapy or corticosteroids. Though B-acute lymphoblastic leukemia can rarely go into spontaneous remission, these patients require close follow-up as most patients will eventually develop recurrence.

Methotrexate-Induced Neurotoxicity in Hispanic Adolescents with High-Risk Acute Leukemia-A Case Series.

Intrathecal methotrexate (IT MTX) and high-dose intravenous methotrexate (HD MTX) are important components of treatment in high-risk acute leukemia. We describe five Hispanic adolescents with high-risk acute leukemia at our institution who experienced MTX-induced neurotoxicity. All patients were eventually rechallenged with MTX. Two of the five patients had a second episode of neurotoxicity, but all patients recovered. Further studies should be performed to determine whether Hispanic patients are more susceptible to MTX neurotoxicity.

Severe anemia in an adolescent male with sickle cell trait: a case report.

BACKGROUND: Sickle cell trait is generally considered a benign condition. However, it has been associated with uncommon comorbidities such as painless gross hematuria secondary to renal papillary necrosis and renal medullary carcinoma. OBSERVATION: We present a 16-year-old African American boy with sickle cell trait and a recent history of prolonged gross hematuria due to renal papillary necrosis. The patient developed severe iron deficiency anemia and required transfusion support. CONCLUSIONS: Although renal papillary necrosis is well-described, it is uncommon in pediatrics and only rarely results in the need for transfusion.

Retinoic acid fails to induce cell cycle arrest with myogenic differentiation in rhabdomyosarcoma.

BACKGROUND: Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children. Current treatment strategies do not cure most children with recurrent or high-risk disease, underlying the need for novel therapeutic approaches. Retinoic acid has been shown to induce differentiation in a variety of cells including skeletal myoblasts and neuroblasts. In the setting of minimal residual disease, retinoic acid improves survival in neuroblastoma, another poorly differentiated childhood tumor. Whether such an approach is useful for rhabdomyosarcoma has not yet been investigated. Several in vitro studies have demonstrated an appreciable effect of retinoic acid on human RMS cellular proliferation and differentiation. PROCEDURE: We assessed the efficacy of ATRA on rhabdomyosarcoma, in vitro and in vivo, using cell lines and xenografts. RESULTS: ATRA slowed RMS cell proliferation, and promoted a more differentiated myogenic phenotype in both alveolar and embryonal RMS cell lines. Treatment of cultured murine myoblasts with retinoids increased Myogenin expression, but did not induce cell cycle arrest. Despite the favorable in vitro effects, ATRA failed to delay relapse of minimal residual disease using human RMS xenografts in immuno-suppressed NOD-SCID (NSG) mice. Interestingly, tumors that recurred after ATRA treatment showed evidence of enhanced muscle differentiation. CONCLUSION: Our results indicate that ATRA could increase the expression of some genes associated with muscle differentiation in rhabdomyosarcoma cells, but there was no benefit of single-agent therapy in an MRD model, likely because cell cycle arrest was uncoupled from the pro-differentiation effects of retinoids.