Comparison of 1-day vs 2-day dosing of high-dose melphalan followed by autologous hematopoietic cell transplantation in patients with multiple myeloma

• Published in: Bone marrow transplantation (Basingstoke) Vol. 49; no. 6; pp. 761 - 766
• Main Authors: Parmar, S R, Bookout, R, Shapiro, J F, Tombleson, R, Perkins, J, Kim, J, Yue, B, Tomblyn, M, Alsina, M, Nishihori, T
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.06.2014; Nature Publishing Group
• Subjects: 631/250/1904; 692/699/67/1059/99; 692/699/67/1990/804; Adult; Aged; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Autografts; Biological and medical sciences; Bone marrow; Bone marrow, stem cells transplantation. Graft versus host reaction; Care and treatment; Cell Biology; Combined Modality Therapy; Comparative analysis; Confidence intervals; Disease-Free Survival; Dosage; Dosage and administration; Drug Administration Schedule; Female; Hematologic and hematopoietic diseases; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Humans; Immunodeficiencies. Immunoglobulinopathies; Immunoglobulinopathies; Immunopathology; Internal Medicine; Kaplan-Meier Estimate; Length of Stay; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; Medicine; Medicine & Public Health; Melphalan; Melphalan - administration & dosage; Melphalan - adverse effects; Middle Aged; Mucositis; Multiple myeloma; Multiple Myeloma - drug therapy; Multiple Myeloma - therapy; Myeloablative Agonists - administration & dosage; Myeloablative Agonists - adverse effects; original-article; Patients; Public Health; Resource utilization; Response rates; Retrospective Studies; Stem cell research; Stem cell transplantation; Stem Cells; Toxicity; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation; Transplantation Conditioning - adverse effects; Transplantation Conditioning - methods; Treatment Outcome; Antineoplastic agent; Human; Immunopathology; Hematology; Stem cell; Hematopoietic cell; Malignant hemopathy; Myeloma; Posology; Alkylating agent; Immunoglobulinopathy; Nitrogen mustard; Lymphoproliferative syndrome; Melphalan; Autologous hematopoietic stem cell transplantation; High dose; Comparative study; Hematopoietic stem cell transplantation; Cancer
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/bmt.2014.56

High-dose melphalan at 200 mg/m 2 can be administered in 1 day or over 2 consecutive days before autologous hematopoietic cell transplantation (HCT) for multiple myeloma (MM). Limited data exist on the comparison of the two dosing schedules. A retrospective study of 278 consecutive MM patients receiving high-dose melphalan from January 2010 to December 2012 was conducted. Objectives were to compare the length of hospitalization, toxicity profile, response rates, PFS and OS. One hundred and eighty five patients received 2-day dosing and 93 received 1-day dosing. The two end points of the 95% confidence interval (CI) for the difference did not exceed the preselected margin, therefore the length of hospitalization was considered equivalent. No significant differences were found for response rates, PFS and OS. The toxicity profile was similar with the exception of more frequent ⩾grade 3 oral mucositis in the 2-day group (13.5% vs 5.4%; odds ratio 3.07 (95% CI:1.11–8.48); P =0.03). High-dose melphalan, given either in 1 day or over 2 days, produced comparable treatment outcomes except for increased grade 3/4 mucositis in the 2-day regimen. One-day dosing could shorten the hospital stay by 1 day and may allow better resource utilization.