Beta-thalassemia Major

Sickle Cell Disease

Iron Hemosiderosis

Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg 3 times/d (better tolerated if started and then built up over 4 weeks).
Dose may be increased up to 100mg/kg/day guided by serum ferritin. Agranulocytosis risk 1-2%. Monitor TLC and granulocytic count / 2weeks. Patients need to be continually educated about this risk, know that they must stop DFP if they have a fever or infection.
Drug: Deferasirox will be orally administered at a dose of 20 mg/kg once daily at evening for 5 days.
Other Name: ICL670

DFP (ferriprox) and deferasirox (ICL 670)

Drug: Deferoxamine It will be administered subcutaneously over 8 hours for 5 days at a dose of 40 mg/kg.
Other Name: Desferal, DFO Drug: DFP DFP: will be orally administered at a dose of 75mg/kg orally in 3 divided doses for 7 days

DFP, DFO

Pediatric Hematology clinic, Ain Shams University

Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload

A Prospective Randomized Comparative Study of Efficacy and Safety of Combined Deferiprone (DFP) and Deferasirox Versus DFP and Desferrioxamine (DFO) Therapy in Diseases With Severe Iron Overload

• The primary outcome measure is to measure the change in serum ferritin level from baseline in the 2 combination therapy.

• The secondary outcome measure is to determine the number of patients who will develop adverse reactions upon administering the drugs in combination.

Mohsen Saleh Elalfy

professour of pediatrics

Ain Shams University

Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H
Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0.
Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor.

Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H
Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0.
Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor.
Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg
Days -9 through -6
Fludarabine: 30mg/m2 Day -5 through Day -2
Campath 1H: Per institutional guidelines Days -5 through -2
Cyclophosphamide: 50 mg/kg Days -5 through -2
MESNA: 10 mg/kg x 5 Days -5 through -2

Bone Marrow or Stem Cell Infusion

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Engraftment is defined as an absolute neutrophil count (ANC) >500/microL x 3 days.

Engraftment Rate After Transplant

Stable hematopoietic chimerism is defined as having 50-99 percent of donor cells.

Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)

Transient mixed hematopoietic chimerism is defined as any mixed chimerism return to 100 percent donor.

Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)

All AEs and SAEs (including infections) will be collected for evaluation of infectious complications.

Number of Participants With Infectious Complications

Hematopoietic: defined as transfusion independence.

Hematopoietic Reconstitution

Immune reconstitution: defined as absolute lymphocyte count (ALC) >1000x10e3/microL

Immune Reconstitution

Acute GVHD is graded by the method of Przepiorka D. et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD

Number of Participants With ACUTE GVHD

Chronic GVHD is graded by NIH guidelines for chronic GVHD, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3 where 0 means no chronic GVHD, and 3 is the highest stage of chronic GVHD.

Number of Participants With CHRONIC GVHD

Event-free survival is calculated from the date of transplant to the date of graft failure, disease recurrence or death from any cause.

Event-free Survival

Overall Study

Dr. Tami D. John

Thalassemia

4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg
Days -9 through -6

Busulfan

Fludarabine

Per institutional guidelines Days -5 through -2

Campath 1H

Cyclophosphamide

MESNA

Texas Children's Hospital

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia

beta-thalassemia/seizures

Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

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