STEM CELL MOBILIZATION WITH HIGH-DOSE PLERIXAFOR IN PATIENTS WITH SICKLE CELL DISEASE

Prior treatment

• Emergency room admission or hospitalization in the past 14 days prior to first dose of study drug Major surgery in the past 30 days prior to first dose of study drug Active and painful splenomegaly or splenomegaly (size greater than upper limit of normal on examination).
• Participant who has received experimental therapy within 4 weeks prior to providing informed consent Poorly controlled diabetes mellitus, as assessed by the Investigator Concomitant treatment with alternative investigational agent unable to be held for 30 days Unwillingness to use a highly effective method of contraception for 1 month after motixafortide Pregnancy Inability or unwillingness of research participant or legal guardian/ representative to give written informed consent.
• Inability or unwillingness of research participant to hold hydroxyurea for 30 days prior to first dose of study drug

Other illness

• Participant who, by medical history, requires rare donor registry RBC units for transfusion, or is unable to receive routine transfusion. Eligible study participants must have undergone prior work-up for the presence of red cell alloantibodies and confirmation of available compatible blood product support Known allergy to or contraindication for motixafortide administration, or medications routinely administered during apheresis Participant who has had a prior autologous or allogeneic transplantation, inclusive of gene therapy Active viral, bacterial, fungal, or parasitic infection. History of cancer, excluding squamous carcinoma of the skin and cervical carcinoma in situ.

• Patients with sickle cell disease 18 to 25 years old At least one allogeneic HSCT indication followin the Brazilian Bone Marrow Transplant Society (SIMÕES et al., \[s.d.\]) ECOG/ Karnofsky/Lansky scores \> 80 Hemoglobin \> 7 g/dL, WBC counts \> 3000/mm3, neutrophil counts \> 1500/mm3, platelet counts \> 150000/mm3 No evidence of severe hepatic disfunction, defined as aspartate aminotransferase and alanine aminotransferase \< 5 times ULN or bilirubin \< 2,5 times ULN No evidence of renal disfunction, defined as creatinine \<1,5 mg/dL ou GFR\> 60 mL/min LVEF \> 40% and no signals of pulmonary hypertension Negative serologies for HIV, HBV, HCV, syphilis, Chagas disease or HTLV Being able to undergo partial exchange transfusion to lower HbS \<30% within one week before CD34+ mobilization and collection No pregnancy or breastfeeding; acceptance to use two contraceptive methods during the study.