Hematopoietic chimerism following immunoablative therapy for non-malignant disorders-out-patient stem cell transplantation

Reggie E Duerst*, Paul R. Haut, Lakshmi Venkateswaran, Morris Kletzel

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


We have treated 4 patients {hemoglobinopathy, n=2 and hyper-IgM immunodeficiency (CD 154 def), n=2 ) utilizing an immunoablative regimen [fludarabine, 30 mg/mA2/d IV x 6 d, busulfan, I mg/kg IV x 8 and anti-thymocyte globulin (ATG), 40 mg/kg/d IV x 4d] followed by unmanipulated peripheral blood SCT from an HLA matched sibling donor. The donors received G-CSF ( 10 mcg/kg/d x 4d) for stem cell mobilization. Oral cyclosporin A was administered for prophylaxis of GVHD. Growth factor support was not routinely administered. Patients were cared for in the Ambulatory Stem Cell Unit. Chimerism was documented by assessment of VNTR or FISH for X-chromosome specific DNA. The patients with CD154def had liver dysfunction related, in part, to prior cryptosporidium infection. Patients received 5.6-7.6 xlOA8 MNC/kg, 6.2-8.1 x 10A6 CD34+ cells/kg. Rapid development of mixed chimerism resulted in minimal need for transfusion support. No red cell transfusions were required post SCT and only one patient required platelet transfusions (x3). Severe neutropenia and mucositis did not develop. Brief hospital admissions (n = 4) following SCT were for neutropenic fever (3) and aseptic meningitis (1). CD40 Hgand expression has increased in CD 154def recipients to -40-50% of CD3+CD8- cells. Acute and chronic GVHD have developed in the 23 yo patient with homozygous sickle hemoglobinopathy. Diagnosis Age (yrs) Days hospitalized Max T Bill (mg/dl) Chimerism F/U S/P SCT post Day "0" (days) Sickle Cell 23 9 2.5 Full 206 Thalassemia 7 0 0.7 Full 52 Hyper-IgM 54 2 Mixed 109 Hyper-IgM 9 5 4.5 Mixed 59 This initial experience indicates immunoablative therapy followed by SCT from matched sibling donors can be administered safely in the outpatient setting for patients with Stem Cell defects. The risk of toxicity for the patient and medical costs are greatly reduced when compared with recipients of myeloablative therapy.

Original languageEnglish (US)
Issue number11 PART II
StatePublished - Dec 1 2000

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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