Cyclophosphamide | Aplastic Anemia and MDS International Foundation

Cyclophosphamide

An alkylating agent, a class of chemotherapy drugs which interfers with the growth of diseased cells. Cyclophosphamide is approved by the U.S. Food and Drug Administration (FDA) for treating certain diseases. It is still an investigational therapy, in combination with other medications, for treating aplastic anemia and other forms of bone marrow failure.
Brand name: 
Cytoxan
Bone Marrow Disease(s): 
aplastic anemia

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Cyclophosphamide (INN), also known as cytophosphane, is a medication mainly used in chemotherapy. It is an alkylating agent of the nitrogen mustard type (specifically, the oxazaphosphorine group).

An alkylating agent adds an alkyl group to DNA. It attaches the alkyl group to the guanine base of DNA, at the number 7 nitrogen atom of the imidazole ring. This interferes with DNA replication by forming intrastrand and interstrand DNA crosslinks.

Cyclophosphamide is used to treat cancers, autoimmune disorders, and AL amyloidosis. As a prodrug, it is converted by liver cytochrome P450 (CYP) enzymes to form the metabolite 4-hydroxy cyclophosphamide that has chemotherapeutic activity.

Cyclophosphamide has severe and life-threatening adverse effects, including acute myeloid leukemia, bladder cancer, hemorrhagic cystitis, and permanent infertility, especially at higher doses. For autoimmune diseases, doctors often substitute less-toxic methotrexate or azathioprine after an acute crisis.

It is on the World Health Organization's List of Essential Medicines, a list of the most important medication needed in a basic health system.

The abbreviation CP is common for referring to cyclophosphamide, but abbreviating drug names is not best practice in medicine.

Related Clinical Trials

Clinical Trial Status Age Group
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies Recruiting 18 years and older
Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy Recruiting up to 75 years
Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation Not yet recruiting 18 years and older
Busulfan and Cyclophosphamide Followed By ALLO BMT Recruiting up to 44 year
Calcineurin Inhibitor-Free Interventions for Prevention of Graft-versus-Host Disease (BMT CTN 1301) Recruiting 1 to 65 years
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant Recruiting up to 22 years
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant Recruiting Up to 22 years
Conditioning Regimen and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematologic Malignancies. Recruiting 18 years to 70 years
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia Recruiting Up to 59 years
Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Recruiting not provided
Donor Stem Cell Transplant With No or Low-Intensity Chemotherapy Using Sirolimus and Treated Immune Cells to Treat Blood and Lymph Cancers Recruiting 11 years to 90 years
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer Recruiting up to 69 years
Donor Umbilical Cord Blood Transplant With or Without Ex-vivo Expanded Cord Blood Progenitor Cells in Treating Patients With Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or Myelodysplastic Syndromes Recruiting 6 months to 45 years
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerou Recruiting Up to 54 years
Fludarabine Phosphate, Cyclophosphamide, Total-Body Irradiation, and Donor Bone Marrow Transplant Followed by Donor Natural Killer Cell Therapy, Mycophenolate Mofetil, and Tacrolimus in Treating Patients With Hematologic Cancer Recruiting
Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia Recruiting 18 and older
GVHD Prophylaxis With Post Transplant Cyclophosphamide for Patients With Renal Insufficiency Undergoing a Conventional 8/8 HLA-matched Related or Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplant Recruiting 18 years or older
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia Recruiting Up to 70 years
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia Recruiting Up to 44 years
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA) Recruiting 3 months and older
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA) Recruiting 3 months and older
Intra-Osseous Co-Transplant of UCB and hMSC Recruiting 18 to 75 years
Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Cyclophosphamide, Total-Body Irradiation and Donor Bone Marrow Transplant in Treating Patients With Advanced Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or High-Risk Myelodysplastic Sy Recruiting 18 years and older
Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases Recruiting up to 55 years
Non-Myeloablative Conditioning for Unrelated Donor Umbilical Cord Blood Transplant Recruiting up to 75 years
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure Recruiting 16 years to 80 years
Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN 1203) Recruiting 18 years to 75 years
Optimizing Cord Blood and Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) (CHAMP) Recruiting up to 75 years
Organ-Sparing Marrow-Targeted Irradiation Before Stem Cell Transplant in Treating Patients With High-Risk Hematologic Malignancies Recruiting 18 years to 75 years
Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Recruiting 18 years to 60 years
Pilot Study of Lupron to Improve Immune Function After Allogeneic Bone Marrow Transplantation Recruiting 9 years to 40 years
Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis Recruiting 19 years to 65 years
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies Recruiting 18 years and older
Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic Diseases Recruiting up to 74 years
Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Malignancies Recruiting 18 years and older
RIC Transplant Using Haplo Donors Recruiting 18 years to 75 years
Selective Depletion of CD45RA+ Naive T Cells From Allogeneic Peripheral Blood Stem Cell Grafts From HLA-Matched Related and Unrelated Donors for Prevention of GVHD Recruiting 14 years to 60 years
Shorter Course Tacrolimus After Nonmyeloablative, Related Donor BMT With High-dose Posttransplantation Cyclophosphamide Recruiting 6 monthgs to 75 years
Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML) Recruiting up to 18 years
Stem Cell Transplantation From HLA Partially-Matched Related Donors for Patients With Hematologic Malignancies Recruiting 2 to 21 years
StemRegenin-1 Expanded vs Unexpanded UCB for High Risk Heme Malignancies Recruiting 2 to 35 years
T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts (MOHEL) Recruiting up to 55 years
T-Cell Replete Haploidentical Donor Hematopoietic Stem Cell Plus Natural Killer (NK) Cell Transplantation in Patients With Hematologic Malignancies Relapsed or Refractory Despite Previous Allogeneic Transplant Recruiting up to 21 years
TBI Dose De-escalation for Fanconi Anemia Recruiting
Total Marrow and Lymphoid Irradiation and Chemotherapy Before Donor Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Leukemia Recruiting 12 years to 60 years
Total Marrow Irradiation for Refractory Acute Leukemia Recruiting up to 55 years
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia Available up to 73 years
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia Recruiting up to 73 years
Umbilical Cord Blood Transplant, Cyclophosphamide, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematologic Disease Recruiting 6 months to 45 years

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any drug, treatment or therapy listed on this website. Some therapies listed on our site are considered experimental for the treatment of bone marrow failure diseases. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding any therapy, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.