1800 Williams St., Suite 200 • Denver, CO 80218
Phone 303-388-4876 • Fax 303-336-2193 • Toll Free 1-800-891-7622

URL http://www.rockymountainbmt.com/clinical_trials/Cyclophosphamide-and-Antithymocyte-Globulin-With-or-Without-Total-Body-Irradiation-Followed-By-Allogeneic-Hematopoietic-stem-Cell-Transplantation-For-Severe-Aplastic-Anemia-37.html

Cyclophosphamide and Antithymocyte Globulin, With or Without Total Body Irradiation, Followed By Allogeneic Hematopoietic stem Cell Transplantation For Severe Aplastic Anemia

Protocol Number: RMBMT-02


Major Objectives

Treatment Plan
  • Cyclophosphamide 50mg/kg/day in the morning of days –5 through –2.
  • ATG 30mg/kg in the evening of days –5 through –3.
  • 12 hours between the am cyclophosphamide dose and the pm ATG dose.
  • TBI 200cGY on Day 0 for unrelated donor transplant recipients.
  • Transplant on Day 0.


Supportive Care
Supportive care will follow Standard Operating Procedures and Institutional Guidelines.

Patient Selection

Inclusion Criteria:

A diagnosis of severe aplastic anemia (SAA) or paroxysmal nocturnal hemogloinuria (PNH with the following criteria:
  • Bone marrow cellularity <20%.
  • Two or more of the following abnormalities: ANC ≤ 500μl, platelets ≤ 20,000/μ/l, reticulocytes ≤ 50,000/μl.
  • Total serum bilirubin < 2 times the upper limit of normal and calculated creatinine clearance ≥ 60ml/minute.
  • Left ventricular ejection fraction measured by radionuclide ventriculography of ≥ 50%.
  • An HLA-compatible donor, defined as a related or unrelated donor who is HLA-identical or a related donr mismatched at a single HLA-locus.

Exclusion Criteria:

Patients will be ineligible for treatment for any of the following reasons:
  • Uncontrolled infection or comorbidities that would preclude transplantation.
  • Female patients who are pregnant and/or who do not practice adequate contraception.
  • Patients who are HIV positive.
  • Patients with clonal cytogenetic abnormalities or myelodysplasia diagnosed less than 3 months prior to the diagnosis of SAA.
  • Patients with congenital or constitutional aplastic anemia or Fanconi anemia.


Exceptions to these inclusion criteria or will be decided upon at the Pipeline meeting and must be approved by the entire group before proceeding to transplant.