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Autologous Hematopoietic Cell Transplantation for Core-binding Factor Positive Acute Myeloid Leukemia in the First Complete Remission

Phase 2
15 Years
65 Years
Open (Enrolling)
Acute Myeloid Leukemia

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Trial Information

Autologous Hematopoietic Cell Transplantation for Core-binding Factor Positive Acute Myeloid Leukemia in the First Complete Remission


5.2.1. PBSCs will be collected during recovery phase after the second cycle of HDAC
consolidation chemotherapy.

5.2.2. For mobilization, recombinant human granulocyte- colony-stimulating factor (G-CSF,
Filgrastim) will be given subcutaneously at a dose of 10 mcg/kg from day 9 of the 2nd HDAC
cycle until the completion of harvesting.

5.2.3. PBSCs harvesting will be performed via perm-cath or Quinton catheter, which will be
inserted with fluoroscopy-guided before starting and will be removed after finishing

5.2.4. Harvest will be initiated from the day when the peripheral absolute neutrophil count
reach up to 0.2 x 109/mm3. Peripheral blood mononuclear cells will be collected by
leukapheresis method with 'CS-3000' (Baxter, Deerfield, IL) or other compatible machine.
Cell collection will be performed for at least 2 days and the duration of collection can be
extended until a target CD34+ cell dose of 5 x 106/kg (in terms of body weight of recipient)
is collected.

5.2.5. If adequate numbers of CD34+ cells are not collected, the 3rd HDAC chemotherapy will
be done and mobilization/harvest procedure will be repeated.

5.2.6. Stem cells harvested from patient will be frozen in liquid nitrogen tank for future
autologous HCT cell infusion.

5.3.PRETRANSPLANT WORKUP FOR AUTOLOGOUS HCT 5.3.1. Pre-workup for checking the current
disease status and feasibility for autologous HCT will be performed before the conditioning
and infusion of autologous HCT.

5.3.2. It is recommended that baseline workup for hematologic / non-hematologic status will
be performed at least 1 month prior to the initiation of conditioning. Bone marrow aspirate/
biopsy with cytogenetics and diagnostic lumbar puncture can be performed immediately before
the initiation of conditioning.

5.3.3. Pretransplant workup tests are listed in Appendix IV.

will be given norethindrone (Norlutate) 10mg po daily until platelet count is consistently
over 100,000 /mcL. body weight (ABW) for busulfan will be calculated using following guidelines; ABW is less or equal to ideal body weight (IBW), use ABW. ABW is
greater than IBW but ABW is within 120% of IBW, use IBW. ABW is 120% or greater
than IBW, use IBW+25% (ABW-IBW). body weight) will be calculated in 50 kg + 2.3kg (height[inch]-60) 45.5kg + 2.3kg
(height[inch]-60) 5.4.2. On day -8, preparation drugs will be
administered to patient as below; with 0.9% normal saline will be started
at 6PM and infused continuously at a rate of 100mL/hr through day -4. 300mg/day will be taken p.o. once a day from day-8 to -2. 100 U/kg/day in normal saline 500 mL will be infused continuously at a rate
of 20mL/hr through day 20 (6pm). Heparin should be discontinued if there is clinically
significant bleeding or aPTT level exceeds 1.2 time of the upper normal limit. 15mg/kg (ABW) in normal saline 300cc will be infused continuously at a rate
of 200mL/hr at 8PM, and then, 200mg will be taken p.o. twice a day through day -4. Dilantin
level should be monitored in the morning of day-7 and -6 to maintain the level within 10 to
20 mg/L.

5.4.3. From day-7, conditioning drug will be administered to patient as below; 3.2mg/kg/day in normal saline of 10 times the volume of busulfan will be
infused continuously over 3 hours with an interval of 24 hours on days -7 to -5 (for 3
days). 200mg/m2/day in normal saline 1000mL will be infused continuously over 5
hours twice a day with an interval of 12 hours on day-3 to -2 (for 2 days). such as serotonin antagonist or lorazepam are allowed.

5.5. AUTOLOGOUS PBSCS INFUSION 5.5.1. Autologous PBSCs will be infused on day0. including pheniramine maleate (avil) 45.5mg i.v., acetaminophen 600mg
p.o., hydrocortisone 250mg i.v. and furosemide 20mg i.v. will be given 30 minutes before
cell infusion. will be infused via saline infusion set without in-line filters.
sign should be checked 4 times with 15 minutes of interval, 4 times with 30 minutes, and
then 4 times with 1 hour. Electrocardiogram monitoring will be performed from the start
until the completion of PBSCs infusion. medications should not be administered simultaneously with PBSCs; total parenteral nutrition fluid or
liposomal amphotericin 5.6. SUPPORTIVE CARE 5.6.1. Recombinant human G-CSF (rhG-CSF) 450㎍
in 100 mL of D5W will be infused intravenously over 30 minutes from day 5 till ANC>3,000/㎕.

5.6.2. Ciprofloxacin 500mg will be given p.o. twice a day (for selective bowel
decontamination) from day 1 till ANC>3,000/㎕. With the first fever spike, ciprofloxacin can
be replaced with broad spectrum antibiotics.

5.6.3. Micafungin 50mg will be infused intravenously once a day from day1 till ANC>3,000/㎕.

5.6.4. Acyclovir 250mg/m2 will be infused twice a day from day1 till ANC>3,000/㎕.

5.6.5. In case of oral mucositis, sodium bicarbonate/saline mouthwash will be applied four
times a day until resolved.

5.6.6. Clotrimazole or Canesten® powder can be applied to groin, axilla, and perianal area
twice a day from day-8 until engraftment.

5.6.7. All cellular blood products must be transfused after leukocyte filtration and

5.6.8. For woman of childbearing potential, adequate consultation for ovarian protection can
be sought. For man who intend to have child(ren) in future, sperm banking can be recommended
and performed.

5.7. EVALUATION DURING TREATMENT 5.7.1. CBC with differential count, ALT/AST, bilirubin
with BUN/Cr, and electrolyte will be checked once daily.

5.7.2. Chemical battery with BUN/phosphorus, LDH and level of magnesium will be checked
three times a week.

5.7.3. Coagulation battery with fibrinogen/d-dimer will be checked at day 0, 7, 14, 21 and
once a week thereafter until discharge.

5.7.4. Protein C, antithrombin III, t-PA antigen, and PAI-1 antigen will be checked at day
0, 7, 14, and 21.

5.7.5. Urinalysis will be checked once a week. 5.7.6. Chest X-ray will be checked once a
week routinely and more frequently according to patient's condition.

5.7.7. Blood galactomannan (aspergillosis antigen, GM) assay can be recommended to be
checked once a week until ANC>3,000/ according to the physician's decision.

5.8. POST-HCT FOLLOW-UP INCLUDING MRD MONITORING 5.8.1. Patients will be followed with
physical examination and appropriate blood test including CBC at least every 3 months for 3
years, every 6 months for the next 2 years, and then annually thereafter.

5.8.2. Toxicities related to treatment will be assessed and reported according to NCI CTCAE

5.8.3. Minimal residual disease (MRD) monitoring with RQ-PCR for AML1/ETO or CBFβ/MYH11 will
be performed before autologous HCT (bone marrow and peripheral blood, 1 month after
autologous HCT (bone marrow and peripheral blood), and thereafter, every 3 months
(peripheral blood only) until 3 years after autologous HCT.

5.8.4. If RQ-PCR is not available, RT-PCR and/or FISH can be used for MRD monitoring

Inclusion Criteria:

- Patients with CBF positive AML in CR1. CBF AML includes t(8;21)(q22;q22)
[AML1(RUNX1)/ETO(CBFα2T1)], inv(16)(q13q22) (CBFβ/MYH11),t(16;16)(p13;q22)
(CBFβ/MYH11) Using RT-PCR, FISH, or standard karyotype analysis technique.

- Patients who plan to receive the second cycle of HDAC consolidation chemotherapy.

- 15 years old or older and 65 years or younger

- Adequate performance status (Karnofsky score of 70 or more).

- Adequate hepatic and renal function (AST, ALT, and bilirubin < 3.0 x upper normal
limit, and creatinine < 2.0 mg/dL).

- Adequate cardiac function (left ventricular ejection fraction over 40% on heart scan
or echocardiography)

- Signed and dated informed consent must be obtained from patient.

Exclusion Criteria:

- Presence of significant active infection

- Presence of uncontrolled bleeding

- Any coexisting major illness or organ failure

- Patients with psychiatric disorder or mental deficiency severe as to make compliance
with the treatment unlike, and making informed consent impossible

- Nursing women, pregnant women, women of childbearing potential who do not want
adequate contraception

- Patients with a diagnosis of prior malignancy unless disease-free for at least 5
years following therapy with curative intent (except curatively treated nonmelanoma
skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:


Outcome Description:

Relapse incidence (cumulative incidence of relapse, CIR) Disease-free survival (DFS)

Outcome Time Frame:

6 years

Safety Issue:


Principal Investigator

Dae-Young Kim, professor

Investigator Role:

Principal Investigator

Investigator Affiliation:

Asan Medical Center


Korea: Food and Drug Administration

Study ID:




Start Date:

January 2010

Completion Date:

December 2015

Related Keywords:

  • Acute Myeloid Leukemia
  • CBF(+)
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid