High-dose Carboplatin/Etoposide/Melphalan increases risk of thrombotic microangiopathy and organ injury after autologous stem cell transplantation in patients with neuroblastoma

Autor: Sonata Jodele, Adam Lane, Gregory Wallace, Brian Weiss, Ashley Teusink-Cross, Kasiani C. Myers, Stella M. Davies, Christopher E. Dandoy
Rok vydání: 2018
Předmět:
Male
Melphalan
medicine.medical_specialty
medicine.medical_treatment
Urology
ThioTEPA
Hematopoietic stem cell transplantation
Antibodies
Monoclonal
Humanized
urologic and male genital diseases
Transplantation
Autologous
Disease-Free Survival
Carboplatin
Neuroblastoma
03 medical and health sciences
0302 clinical medicine
Autologous stem-cell transplantation
immune system diseases
hemic and lymphatic diseases
Antineoplastic Combined Chemotherapy Protocols
medicine
Humans
Child
neoplasms
Etoposide
Retrospective Studies
Transplantation
Chemotherapy
Thrombotic Microangiopathies
business.industry
Hematopoietic Stem Cell Transplantation
Hematology
female genital diseases and pregnancy complications
Survival Rate
Regimen
Child
Preschool
030220 oncology & carcinogenesis
Female
business
030215 immunology
medicine.drug
Zdroj: Bone Marrow Transplantation. 53:1311-1318
ISSN: 1476-5365
0268-3369
DOI: 10.1038/s41409-018-0159-8
Popis: Transplant-associated thrombotic microangiopathy (TA-TMA) is an increasingly recognized complication of hematopoietic cell transplant that can result in multi-organ failure (MOF). Patients undergoing high-dose chemotherapy with autologous stem cell transplant (aHCT) for neuroblastoma require good organ function to receive post-transplant radiation and immunotherapy. We examined TA-TMA incidence and transplant outcomes in patients with neuroblastoma receiving different transplant preparative regimens. Sixty patients underwent aHCT using high-dose chemotherapy: 41 patients received carboplatin/etoposide/melphalan (CEM), 13 patients busulfan/melphalan (Bu/Mel) and six patients received tandem transplant (cyclophosphamide/thiotepa and CEM). TA-TMA with MOF was diagnosed in 13 patients (21.7%) at a median of 18 days after aHCT. TA-TMA occurred in 12 patients receiving CEM and in 1 after cyclophosphamide/thiotepa. There were no incidences of TA-TMA after Bu/Mel regimen. Six of 13 patients with TA-TMA and MOF received terminal complement blocker eculizumab for therapy. They all recovered organ function and received planned post-transplant therapy. Out of seven patients who did not get eculizumab, two died from TA-TMA complications and four progressed to ESRD. We conclude that the CEM regimen is associated with a high incidence of clinically significant TA-TMA after aHCT and eculizumab can be safe and effective treatment option to remediate TA-TMA associated MOF.
Databáze: OpenAIRE
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