scholarly journals MBCL-29. PHASE I/II STUDY OF SEQUENTIAL HIGH-DOSE CHEMOTHERAPY WITH STEM CELL SUPPORT IN CHILDREN YOUNGER THAN 5 YEARS OF AGE WITH HIGH-RISK MEDULLOBLASTOMA

2020 ◽  
Vol 22 (Supplement_3) ◽  
pp. iii394-iii395
Author(s):  
Christelle Dufour ◽  
Julien Masliah-Planchon ◽  
Marie-Bernadette Delisle ◽  
Anne Geoffray ◽  
Rachid Abbas ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Phase I ◽  
Complete Response ◽  
Multicenter Trial ◽  
High Dose Chemotherapy ◽  
Craniospinal Irradiation ◽  
High Dose ◽  
Stem Cell Rescue

Abstract PURPOSE To assess the 3-year EFS rate of children younger than 5 years of age with high-risk medulloblastoma (MB) treated according to the prospective multicenter trial HR MB-5. PATIENTS AND METHODS After surgery, all children received 2 cycles of Etoposide- Carboplatine. If partial (PR) or complete response (CR) was achieved after induction chemotherapy, children received 2 courses of thiotepa (600mg/m²) with stem cell rescue. For patients in CR after high-dose chemotherapy, they received one course of Cyclophosphamide – Busilvex with stem cell rescue (Phase I part). The others patients (not in PR after induction or in CR after thiotepa) were treated with 2 cycles of Temozolomide-Irinotecan followed by age-adapted craniospinal irradiation and maintenance treatment. RESULTS 28 children (2 to 4 years; median: 3.0 years) were enrolled. Group 3 MB were most common (57%). The response rate to Etoposide-Carboplatine was 60.7%. Among 20 patients treated with Thiotepa, 13 children were in CR and received Cyclophosphamide – Busilvex without radiotherapy. Out of them, 9 patients (45%) are alive in CR without craniospinal irradiation (median follow-up 5 years). Among 15 patients treated with radiotherapy, 8 patients are alive (median follow-up 3.8 years). The study was prematurely stopped for an excess of events. The median follow-up was 4 years (range 1.5 - 6.1). The 3-year EFS and OS were 42.3% [25.9 - 60.6] and 71.3% [52.7 - 84.7], respectively. CONCLUSIONS This risk-adapted strategy did not improve EFS in young children with high-risk MB. However, the study shows that good responders to chemotherapy can be cured without recourse to irradiation.

NCOG-36. LONG-TERM SURVIVAL AND COGNITIVE FUNCTION FOLLOW UP OF PRIMARY CNS LYMPHOMA TREATED WITH R-MVP FOLLOWED BY HIGH DOSE CHEMOTHERAPY WITH AUTOLOGOUS STEM CELL TRANSPLANT CONSOLIDATION

2021 ◽  
Vol 23 (Supplement_6) ◽  
pp. vi159-vi160
Author(s):  
Kate Therkelsen ◽  
Christian Grommes
Keyword(s):  
Stem Cell ◽  
Complete Response ◽  
High Dose Chemotherapy ◽  
High Dose ◽  
Consolidation Therapy ◽  
Term Survival ◽  
Stem Cell Rescue ◽  
Long Term Follow Up

Abstract BACKGROUND Primary central nervous system lymphoma (PCNSL) is a rare central nervous system malignancy, and long-term follow up studies are uncommon. First line therapy is based on high-dose methotrexate and different consolidation therapy options. This is a long-term follow up study of PCNSL patients enrolled in a prospective trial using R-MPV chemotherapy regimen followed by high dose chemotherapy and autologous stem cell rescue to determine long-term survival and cognitive effects. METHODS From June 2005 to September 2011, 32 newly diagnosed immunocompentent PCNSL were enrolled. Patients received 5-7 doses of rituximab (500mg/m2), methotrexate (3.5 gm/m2), procarbazine (100mg/m2), and vincristine (1.4mg/m2) (R-MVP). Consolidation therapy consisted of high dose chemotherapy (HDC) with thiotepa (250 mg/m2), busulfan (3.2 mg/kg), and cyclophosphamide (60 mg/kg), followed by autologous stem cell rescue (ASCT) in those with partial or complete response to R-MVP. Long-term follow-up status including disease status, cognitive status (KPS, NANO score), and leukoencephalopathy (modified Fazkas Scale) were collected. RESULTS 26 of 36 underwent HDC and ACST. Of those, 3 died due to treatment related effects; 2 died of disease progression within two years after ASCT. After a median follow-up of 10.5 years, none of the remaining 21 patients progressed. At the time of last follow up, the median KPS was 90, compared to 80 at time of ASCT. The median NANO score and leukoencephalopathy score post ASCT and at follow-up did not change. Of note, 2 of 4 patients with a partial or complete response to R-MVP that elected not to proceed with HDC-ASCT consolidation, experienced progression at a mean of 52 months. CONCLUSION Long-term follow up demonstrates that treatment was tolerated well with stable leukoencephalopathy on MRI and good performance status. Disease recurrence 2 years after HDC with ASCT consolidation was not observed.

Phase I/II study incorporating intravenous hydroxyurea into high-dose chemotherapy for patients with primary refractory or relapsed and refractory intermediate-grade and high-grade malignant lymphoma.

1995 ◽  
Vol 13 (5) ◽  
pp. 1089-1095 ◽  
Author(s):  
W P Vaughan ◽  
E Kris ◽  
J Vose ◽  
P J Bierman ◽  
P Gwilt ◽  
...  
Keyword(s):  
Phase I ◽  
Continuous Infusion ◽  
Progression Free Survival ◽  
High Dose Chemotherapy ◽  
Rank Test ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
Stem Cell Rescue

PURPOSE A phase I/II study was performed to evaluate the incorporation of hydroxyurea (HU) into high-dose chemotherapy of non-Hodgkin's lymphoma (NHL). PATIENTS AND METHODS Thirty-eight patients with primary refractory and refractory relapsed NHL were treated with carmustine (BCNU) (300 mg/m2 on day -8), cyclophosphamide (Cy) (2.5 g/m2/d on days -8 and -7), etoposide (E) (150 mg/m2 every 12 hours on days -6, -5, and -4), and HU (BCHE) with autologous hematopoietic stem-cell rescue. Twenty-one patients received HU in a dose escalation of 2 to 12 g/m2 intravenously (IV) by 72-hour continuous infusion. When the IV formulation was not available, 17 patients were given 18 g/m2 of HU orally in divided doses every 6 hours over the same 72-hour period. RESULTS The dose-limiting toxicity of 72-hour continuous infusion HU in this regimen was mucositis. Endotracheal intubation was necessary to protect the airway in two thirds of patients treated at 12 g/m2. Six patients (oral BCHE, five of 17; IV BCHE, one of 21) died with nonresponding or progressive disease and, at least in part, from the complications of the high-dose chemotherapy. Seventeen patients (45%) achieved complete remission (CR). More patients treated with IV BCHE achieved CR than patients treated with oral BCHE (12 of 21 v five of 17; P < .1, chi 2 test). Nine patients (two of 17 oral BCHE and seven of 21 IV BCHE) remain disease-free as of January 31, 1994, with a minimum follow-up time of 3 years. The lower mortality and higher response rate with IV BCHE translated into a significantly superior probability of progression-free survival (PFS) (33% at 4 year v 12% for oral BCHE; P = .048, log-rank test). CONCLUSION High-dose BCHE is effective treatment for primary refractory and refractory relapsed NHL. Continuous IV HU appears to be less toxic and more effective than intermittent oral HU in this regimen.

scholarly journals Acute Complications After High-Dose Chemotherapy and Stem-Cell Rescue in Pediatric Patients With High-Risk Neuroblastoma Treated in Countries With Different Resources

2018 ◽  
pp. 1-12 ◽  
Author(s):  
Mahmoud M. Elzembely ◽  
Julie R. Park ◽  
Khaled F. Riad ◽  
Heba A. Sayed ◽  
Navin Pinto ◽  
...  
Keyword(s):  
United States ◽  
Stem Cell ◽  
High Risk ◽  
The United States ◽  
High Dose Chemotherapy ◽  
High Dose ◽  
Stem Cell Rescue ◽  
Resource Limited ◽  
Comparative Review ◽  
Hepatitis Infection

Purpose High-dose chemotherapy with autologous stem-cell rescue (SCR) is a key component of high-risk neuroblastoma (HRNB) therapy. Carboplatin, etoposide, and melphalan (CEM) or busulfan and melphalan (Bu/Mel) are the most evaluated, effective high-dose chemotherapy for HRNB on the basis of results from major cooperative group studies. Toxicity profiles vary between these regimens, and practice variation exists regarding the preferred high-dose therapy (HDT). We sought to evaluate the safety of HDT and autologous SCR for HRNB in a resource-limited country (Egypt) compared with the resource-rich United States. Patients and Methods We performed a retrospective comparative review of single CEM-based HDT/SCR outcomes through day 100 for HRNB at the Fred Hutchinson Cancer Research Center (FH) in the United States (2005 to 2015) versus Bu/Mel-based HDT at El-Sheikh Zayed Specialized Hospital (SZ) in Egypt (2009 to 2015). Results Forty-four patients at FH and 77 patients at SZ were reviewed. Pretransplant hepatic comorbidities were significantly higher at SZ (29 of 77 v nine of 44; P = .05), with 19 of 77 patients at SZ having hepatitis infection. Engraftment was delayed after SZ-Bu/Mel therapy compared with FH-CEM therapy for neutrophils (median 12 days v 10 days, respectively; P < .001) and platelets (median 20 days v 18 days, respectively; P < .001). Sinusoidal obstruction syndrome occurred later, after SZ-Bu/Mel therapy (median 19 days v 7 days; P = .033), and four of eight cases were fatal (six of eight patients had underlying hepatitis infection), whereas three of three cases after FH-CEM therapy were moderately severe. Resource utilization associated with the number of days with fever, antibiotic use, and the number of transfusions administered was significantly higher after FH-CEM therapy than after SZ-Bu/Mel therapy. Conclusion Use of autologous stem-cell transplantation is feasible in the context of a resource-limited country.

High-Dose Chemotherapy and Autologous Stem Cell Transplantation In Patients With High-Risk Neuroblastoma : Results Of Turkish Pediatric Oncology Group- Neuroblastoma 2003 (TPOG-NBL 2003) Protocol

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 3411-3411 ◽  
Author(s):  
Serap Aksoylar ◽  
Ali Varan ◽  
Canan Vergin ◽  
Volkan Hazar ◽  
Ferhan Akici ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Induction Chemotherapy ◽  
Conflicts Of Interest ◽  
Survival Rates ◽  
High Dose Chemotherapy ◽  
High Dose ◽  
Intention To Treat ◽  
Stem Cell Rescue ◽  
Chemotherapy Patients

Abstract Introduction TPOG-NBL 2003 national protocol was designed to improve treatment results of the high risk patients by adjunction of stem cell rescue with intensive multimodal therapy. Material and Methods High risk stratification was made according to COG criteria. Just before the third cycle of chemotherapy, patients without progression were allocated into two treatment groups non-randomly by physicians’ and/or parent’s choices guided to the center’s facility, toxicity and social-economical facility to attain the megatherapy. After an induction of 6 chemotherapy cycles, the protocol was divided into two arms which were designed to continue the intensive conventional chemotherapy (CCT), or initiate myeloablative therapy with autologous stem-cell rescue (ASCR). All patients were also given 13-cis-retinoic acid as maintenance therapy. Results Fifty-six percent (272 patients) of all neuroblastoma patients was evaluated as high risk. Response rate to induction chemotherapy was 81% (CR/VGPR: 32%, PR: 49%) in patients at the end of induction chemotherapy. Overall EFS and OS at 3-years were 36% and 45%, respectively. Intention-to-treat analysis documented post-induction (after the six cycles of induction chemotherapy) EFS of 46% in CCT arm (137 patients) and 37% in ASCR group (55 patients) (p= 0.037); whereas, OS was 59% and 43%, respectively (p=0.052). Thirty-one patients (11%) died of treatment-related complications. Conclusion Survival rates of high-risk neuroblastoma have improved over the last decade in Turkey. The main problems when managing these patients were an effective local control, early progression and death. Megatherapy has not augmented the therapeutic end point in our country’s circumstances. However; the better the supportive care and the higher the patients’ compliance is attained, the higher the survival rates might be obtained in Turkish neuroblastoma patients. Disclosures: No relevant conflicts of interest to declare.

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