Therapeutic uses of stem cells

Exemplary indications for cord blood transplantations (haematopoietic stem cells) in children

The following table illustrates exemplary indications for cord blood transplantation in children.

 

Autologous cord blood transplantation – of one’s own blood.

Allogenic cord blood transplantation – from siblings.

Allogenic cord blood transplantation – from a matched unrelated donor.

Allogeneic cord blood transplantation – from an unmatched unrelated donor.

1. Leukaemias

Acute lymphoblastic leukaemia

no

yes

yes

in clinical trials

Acute myeloid leukaemia

depending on the extent or  progression of the disease

yes

yes

depending on the extent or  progression of the disease

Chronic myeloid leukaemia

no

yes

yes

in clinical trials

2. Hyperplastic disorders of the lymphatic system

Acute lymphogranuloma (Hodgkin’s disease)

depending on the stage of the disease

in clinical trials

in clinical trials

no

Malignant lymphomas

depending on the stage of the disease

depending on the stage of the disease

depending on the stage of the disease

no

3. Myelodysplastic syndrome

Myelogenous chronic leukaemia

no

yes

yes

in clinical trials

Refractory anaemia

no

yes

yes

in clinical trials

Refractory anaemia with sideroblasts

no

yes

yes

in clinical trials

Refractory anaemia with excess blasts

no

yes

yes

in clinical trials

Refractory anaemia with excess blasts in transformation

no

yes

yes

in clinical trials

4. Diseases caused by stem cell defect

Severe aplastic anaemia

yes

yes

yes

in clinical trials

Fanconi’s anaemia

no

yes

yes

in clinical trials

5. Other forms of neoplasia

Embtyonal sympathicoblastoma

yes

in clinical trials

no

no

Wilms’ tumour (kidney tumour)

in clinical trials

no

no

no

Brain tumour

yes

no

no

no

Malignant retinoblastoma

yes

no

no

no

Bone tumour (Ewing’s tumour)

yes

in clinical trials

no

no

6. Autoimmune diseases

Juvenile rheumatoid arthritis

in clinical trials

no

no

no

Systemic lupus erythematosus

in clinical trials

no

no

no

Crohn’s disease

in clinical trials

no

no

no

Evan’s syndrome

in clinical trials

no

no

no

7. Mucopolisacharidosis

Hurler’s syndrome- MPS IH

no

yes

yes

in clinical trials

Maroteaux – Lamy syndrome –MPS
VI

no

in clinical trials

in clinical trials

in clinical trials

8. Inherited red blood cell disorder

Beta – thalassemia

no

yes

in clinical trials

no

Diamond - Blackfan anaemia

no

yes

in clinical trials

no

Sickle cell anaemia

no

yes

in clinical trials

no

9. Inherited immune system disorders

Ataxia – telangiectasia

no

yes

yes

yes

Bare lymphocyte syndrome

no

yes

yes

yes

Common variable immunodeficiency

no

yes

yes

yes

DiGeorge’s syndrome

no

yes

yes

yes

Kostmann’s syndrome

no

yes

yes

yes

Leukocyte adhesion deficiency

no

yes

yes

yes

Hyper – IgM syndrome

no

yes

yes

yes

Omenn syndrome

no

yes

yes

yes

Wiskott – Aldrich syndrome

no

yes

yes

yes

X-linked lymphoproliferative disease

no

yes

yes

yes

Severe combined immunodeficiency (SCID)

no

yes

yes

yes

B-cell and T-cell negative SCID

no

yes

yes

yes

T-cell negative and B-cell positive SCID

no

yes

yes

yes

SCID with adenosine deaminase deficiency

no

yes

yes

yes

10. Other inherited diseases

Cartilage-hair hypoplasia

no

yes

yes

yes

Glanzmann’s thrombasthenia

no

yes

yes

yes

Lesch – Nyhan syndrome

no

yes

yes

yes

Osteopetrosis (marble bone disease)

no

yes

yes

yes

11. Liposomal storage diseases

Adrenoleukodystrophy

no

yes

yes

in clinical trials

12. Diseases treated with hematopoietic stem cell transplantation as an unconventional therapy

Type 1 diabetes

yes

in clinical trials

Paediatric Cerebral palsy

yes

in clinical trials

Tetraplegia

no

in clinical trials

Anoxic brain injury

yes

in clinical trials

Accidental brain injury

yes

in clinical trials

Exemplary indications for cord blood transplantation (haematopoietic stem cells) in adults

The following table illustrates exemplary indications for cord blood transplantation in adults.

 

Autologous cord blood transplantation – of one’s own blood.

Allogenic cord blood transplantation – from siblings.

Allogenic cord blood transplantation – from a matched unrelated donor.

Allogeneic cord blood transplantation – from an unmatched unrelated donor.

1. Leukaemias

Acute lymphoblastic leukaemia

in clinical trials

yes

yes

in clinical trials

Acute myeloid leukaemia

depending on the extent or  progression of the disease

yes

in clinical trials

in clinical trials

Chronic myeloid leukaemia

in clinical trials

yes

yes

in clinical trials

2. Hyperplastic disorders of the lymphatic system

Acute lymphogranuloma (Hodgkin’s disease)

depending on the stage of the disease

in clinical trials

in clinical trials

in clinical trials

Malignant lymphomas

depending on the stage of the disease

in clinical trials

in clinical trials

no

3. Myelodysplastic syndrome

Refractory anaemia-RA

in clinical trials

yes

yes

in clinical trials

Refractory anaemia with excess blasts-RAEB

in clinical trials

yes

yes

in clinical trials

Refractory anaemia with excess blasts in transformation RAEBt

in clinical trials

yes

in clinical trials

in clinical trials

4. Plasma cell abnormalities

Multiple myeloma

yes

in clinical trials

in clinical trials

no

5. Diseases caused by stem cell defect

Severe aplastic anaemia

no

yes

yes

in clinical trials

Paroxysmal nocturnal haemoglobinuria

no

yes

in clinical trials

in clinical trials

6. Other forms of cancer:

Germ cell tumours

yes

no

no

no

Breast cancer

in clinical trials

in clinical trials

in clinical trials

no

Ovarian cancer

in clinical trials

in clinical trials

in clinical trials

no

Small cell lung cancer

in clinical trials

no

no

no

Kidney cancer

no

in clinical trials

in clinical trials

no

Glioblastoma – central nervous system cancer

in clinical trials

no

no

no

Reumathoid arthritis

in clinical trials

no

no

no

Multiple sclerosis

in clinical trials

in clinical trials

no

no

Systemic lupus erythematosus

in clinical trials

in clinical trials

no

no

Crohn’s disease

in clinical trials

no

no

no

7. Other diseases

Systemic sclerosis

in clinical trials

in clinical trials

no

no