Background: The clinical course of sickle cell anemia (SCA) is variable, with chronic hemolysis and end-organ damage caused by microvascular occlusion. We evaluated the efficacy and safety of thalidomide plus hydroxyurea (HU) compared with HU alone to determine whether the combination provides a superior clinical benefit and safety profile. Methods: This was an open-label quasi-experimental clinical trial (Clinical Trials Registry of India, CTRI Registration Number 2023/04/065682). Patients with SCA aged > 12 years and postmenopausal females aged > 45 years were allocated 1:1 to receive either HU (20 mg/kg/day) and thalidomide (50 mg/day) in Group A or HU (20 mg/kg/day) only in Group B. Results: The frequency of vaso-occlusive crises (VOCs), transfusion requirements, variations in hematological parameters (hemoglobin [Hb], fetal hemoglobin [HbF], and sickle hemoglobin [HbS]), and side effects between the groups were assessed over 12 months. Repeated-measures analysis of variance was used to determine changes across the observation period. The mean age of the 66 patients diagnosed with SCA (homozygous HbS mutation) was 32.9 (standard deviation ± 11.5) years, and 57.6% were males. Over the 12-month observation period, Group A had significantly fewer VOCs (3.48 ± 2.81) and packed red blood cell transfusions (3.61 ± 2.19) than Group B (11.36 ± 4.20 VOCs; 13.27 ± 3.70 transfusions) (p = 0.0001). There was a significant increase in Hb (8.2 ± 1.8 to 11.8 ± 1.2 g/dL), a decrease in HbS% (72.5 ± 5.5 to 64.5 ± 5.4), and a rise in HbF% (18.9 ± 5.1 to 28.4 ± 5.6) (p < 0.0001) in Group A. Conclusion Combining thalidomide with HU significantly reduced VOCs and transfusion requirements, improved Hb and HbF%, and decreased HbS levels.

Background: This study analyzed the epidemiology and treatment outcomes of germ cell tumorpatients at a single institution. Methods: A retrospective analysis was conducted on intracranial germ cell tumor (iGCT) pa-tients treated at a single tertiary hospital from 2004 to 2019. Patients were categorized based on treatment modality: Korean Society for Pediatric Neuro-Oncology (KSPNO) protocol or bleomycin, etoposide, and cisplatin with radiation therapy. Results: Forty-nine iGCT patients treated with combined chemotherapy and radiotherapywere analyzed. The median age was 19 years (range: 6–40), with a median follow-up duration of 148.0 months (range: 10.5–265.5). Tumors were most common in the pineal gland (51.0%). Although no significant differences in outcomes were observed between treatment modalities, outcomes varied significantly by pathological type. The 10-year progression-free survival rates for germinoma and non-germinomatous germ cell tumors (NGGCTs) were 88.1% and 32.7%, respectively (p=0.003), while the 10-year overall survival rates were 92.9% and 67.5%, respectively (p<0.001). Fourteen patients experienced CTCAE (Common Terminology Criteria for Adverse Events) grade ≥3 adverse events, with one eventrelated death. Conclusion Pure germinoma demonstrated higher survival and lower recurrence rates comparedto NGGCT. The KSPNO protocol appears to be an acceptable and safe treatment option for iGCT patients. Further multi-institutional studies with larger cohorts are warranted.

Background: The clinical course of sickle cell anemia (SCA) is variable, with chronic hemolysis and end-organ damage caused by microvascular occlusion. We evaluated the efficacy and safety of thalidomide plus hydroxyurea (HU) compared with HU alone to determine whether the combination provides a superior clinical benefit and safety profile. Methods: This was an open-label quasi-experimental clinical trial (Clinical Trials Registry of India, CTRI Registration Number 2023/04/065682). Patients with SCA aged > 12 years and postmenopausal females aged > 45 years were allocated 1:1 to receive either HU (20 mg/kg/day) and thalidomide (50 mg/day) in Group A or HU (20 mg/kg/day) only in Group B. Results: The frequency of vaso-occlusive crises (VOCs), transfusion requirements, variations in hematological parameters (hemoglobin [Hb], fetal hemoglobin [HbF], and sickle hemoglobin [HbS]), and side effects between the groups were assessed over 12 months. Repeated-measures analysis of variance was used to determine changes across the observation period. The mean age of the 66 patients diagnosed with SCA (homozygous HbS mutation) was 32.9 (standard deviation ± 11.5) years, and 57.6% were males. Over the 12-month observation period, Group A had significantly fewer VOCs (3.48 ± 2.81) and packed red blood cell transfusions (3.61 ± 2.19) than Group B (11.36 ± 4.20 VOCs; 13.27 ± 3.70 transfusions) (p = 0.0001). There was a significant increase in Hb (8.2 ± 1.8 to 11.8 ± 1.2 g/dL), a decrease in HbS% (72.5 ± 5.5 to 64.5 ± 5.4), and a rise in HbF% (18.9 ± 5.1 to 28.4 ± 5.6) (p < 0.0001) in Group A. Conclusion Combining thalidomide with HU significantly reduced VOCs and transfusion requirements, improved Hb and HbF%, and decreased HbS levels.

Background: This study analyzed the epidemiology and treatment outcomes of germ cell tumorpatients at a single institution. Methods: A retrospective analysis was conducted on intracranial germ cell tumor (iGCT) pa-tients treated at a single tertiary hospital from 2004 to 2019. Patients were categorized based on treatment modality: Korean Society for Pediatric Neuro-Oncology (KSPNO) protocol or bleomycin, etoposide, and cisplatin with radiation therapy. Results: Forty-nine iGCT patients treated with combined chemotherapy and radiotherapywere analyzed. The median age was 19 years (range: 6–40), with a median follow-up duration of 148.0 months (range: 10.5–265.5). Tumors were most common in the pineal gland (51.0%). Although no significant differences in outcomes were observed between treatment modalities, outcomes varied significantly by pathological type. The 10-year progression-free survival rates for germinoma and non-germinomatous germ cell tumors (NGGCTs) were 88.1% and 32.7%, respectively (p=0.003), while the 10-year overall survival rates were 92.9% and 67.5%, respectively (p<0.001). Fourteen patients experienced CTCAE (Common Terminology Criteria for Adverse Events) grade ≥3 adverse events, with one eventrelated death. Conclusion Pure germinoma demonstrated higher survival and lower recurrence rates comparedto NGGCT. The KSPNO protocol appears to be an acceptable and safe treatment option for iGCT patients. Further multi-institutional studies with larger cohorts are warranted.