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Diseases treated by hematopoetic cell transplant

Using the hematopoetical STEM cells from cord blood is something new. They are mainly used as an alternative to the hematopoetic bone marrow which contains adult hematopoetic STEM cells..

Diseases treated with hematopoietic stem cell transplantation:
The first hematopoietic stem cell transplantation performed successfully was achieved 42 years ago (1968), by Dr. Robert A. Good, University of Minnesota. A new therapy age was thus opened for severe hematological diseases that had been considered as incurable until then. Current standard indications for performing a hematopoietic stem cell transplantation include over 70 diseases, most of them malignant and non malignant hematological diseases, but also certain genetic diseases or malignant solid tumors.

Hematopoietic stem cells existing in the bone marrow are the resource of hematopoiesis. Both hematopoietic stem cells and the progenitors (intermediate stages of cell specialization) under the influence of various factors (e.g. genetic factors, environmental factors, infections, etc.) may be affected, resulting in a number of diseases.
These factors can work towards stimulating uncontrolled proliferation of cells belonging to one or more blood cell lines (hematological malignancies e.g. Leukemia) or towards inhibiting the activity of bone marrow hematopoiesis (stem cell diseases with bone marrow failure e.g. Aplastic anemia ).
Another category of non-malignant diseases with indication of making a stem cell transplant, due to severity of evolution and prognosis are hereditary blood diseases affecting the quality or function of blood cells (e.g. in hemoglobinopathies red blood cells are affected; in chronic granulomatosis- neutrophils in immune deficits are affected – white blood cells affected populations) and hereditary metabolic diseases (e.g. Gaucher's disease, mucolipidosis).
For these affections the hematopoietic stem cells graft has a curative, healing role, in some cases being the only saving therapy means. Hematopoietic stem cell transplantation (blood cell maker) is to replace diseased cells with healthy cells after the former were destructed by prior chemoradiotherapy.
Of the category of standard indications of stem cell transplantation, leukemia is the most common malignancy of children, representing about 1 / 3 of the total number of cases of malignancy found in children, followed by brain tumors and Hodgkin's disease.

In Romania it is estimated that 1,500 children suffer from leukemia, acute leukemia representing 10% of common cancers in this age category and the leading cause of death in 35 years. (Acute lymphoblastic leukemia is the most common hematological malignancy arising in childhood, about 80-90% of cases appear under the age of 10 years).
Add to these, 3500 patients with Hodgkin's disease, some of these having the indication to undergo a hematopoietic stem cell transplantation for survival and 3.700 children with thalassemia major, an
inherited blood disease in which the only cure is the transplantation of hematopoietic stem cells from a healthy donor.

Stem cell transplantation may be autologous or allogeneic.(see details above). If the disease has genetic determinism (is caused by an alteration of genetic information), only allogeneic transplantation (transplantation from another healthy, immunologically compatible individual) which should bring "healthy” genetic information (e.g. immunological congenital deficits or congenital metabolic diseases). can play a curative "healer" role.

We know a large number of such diseases (see table), but they have an incidence (frequency) very small population. But if the disease has no such genetic determinism, both autologous transplantation and allogeneic transplantation may be curative role.

Stem cell transplantation may also play only the adjuvant role (complementary) to the fund treatment of some solid malignant tumors. In these cases, the availability of a stored graft and the chance to perform the stem cell transplant allows a therapeutic reassessment of the patient.
Thus it is possible to apply a more aggressive anti-tumor treatment, compared to the usual one (with higher doses of chemo / radiotherapy), aiming to reduce metastasis or tumor recurrence. 
This aggressive treatment is accompanied by bone marrow toxic effects (destruction of bone marrow cells); therefore further administration of a hematopoietic stem cell grafts, which should thus provide bone marrow cell repopulation (e.g. malignant solid tumors: neuroblastoma, retinoblastoma) is required.

Disease

Autolog Transplant

Alogen Transplant
(from donor)

ACUTE LEUKEMIAS

 

 

Acute Lymphoblastic Leukemia (ALL)

Yes *

Yes

Acute Myelogenous Leukemia (AML)

Yes

Yes

Acute Biphenotypic Leukemia

Yes

Yes

Acute Undifferentiated Leukemia

Yes

Yes

CHRONIC LEUKEMIAS

 

 

Chronic Myelogenous Leukemia (CML)

Yes #

Yes

Chronic Lymphocytic Leukemia (CLL)

Yes

Yes

Juvenile Chronic Myelogenous Leukemia (JCML)

Yes #

Yes

Juvenile Myelomonocytic Leukemia (JMML)

Yes #

Yes

LYMPHOPROLIFERATIVE DISORDERS

 

 

Hodgkin’s Disease

Yes

Yes

Non Hodgkin’s Lymphoma

Yes

Yes

Burkitt’s Lymphoma

Yes

Yes

MYELODYSPLASTIC SYNDROMES

 

 

Refractory Anemia (RA)

Yes#

Yes

Refractory Anemia with Excess Blasts (RAEB-T)

Yes#

Yes

Refractory Anemia with Ringed Sideroblasts(RARS)

Yes#

Yes

Refractory Anemia with Excess Blasts

Yes#

Yes

Chronic Myelomonocytic Leukemia (CMML)

Yes#

Yes

MIELOPROLIFERATIV DISEASES

 

 

Myeloid Metaplasia

Yes #

Yes

Agnogenic Myeloid Metaplasia with Myelofibrosis

Yes #

Yes

MONOCLONAL GAMMOPATHY

 

 

Multiple Myeloma

Yes @

Yes

Plasma cell leukemia

Yes @

Yes

Waldestrom ‘s Macroglobulinemia

Yes @

Yes

 

 

 

INHERITED ERYTHROCYTE ABNORMALITIES

 

 

Beta Thalassemia  Major

No

Yes

Blackfan-Diamond Anemia

No

Yes

Pure Red Cell Aplasia

No

Yes

Sickle Cell Disease

No

Yes

STEM CELL DISORDERS

 

 

Aplastic Anemia (severe)

Yes !

Yes

Congenital Cytopenia

No

Yes

Congenital Dyskeratosis

No

Yes

Fanconi Anemia

No

Yes

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Yes

Yes

PHAGOCYTE DISORDERS

 

 

Chediak-Higashi Syndrome

No

Yes

Chronic Granulomatous Disease

No

Yes

Reticular Dysgenesis

No

Yes

Neutrophil Actin Deficiency

No

Yes

LIPOSOMAL STORAGE DISEASES

 

 

Adrenoleukodystrophy

No

Yes

Gaucher’s Disease

No

Yes

Hunter ‘s Syndrome (MPS –II)

No

Yes

Hurler’s Syndrome  (MPS- IH)

No

Yes

Krabbe Disease

No

Yes

Maroteaux-Lamy  Syndrome (MPS – VI)

No

Yes

Metachromatic Leucodystrophy

No

Yes

Morquio Syndrome (MPS – IV)

No

Yes

Mucolipidosis  II (I – cell disease)

No

Yes

Niemann-Pick Disease

No

Yes

San Filipp  Syndrome (MPS III)

No

Yes

Scheie Syndrome (MPS – IS)

No

Yes

Sly Sindrome (MPS – VII)

No

Yes

Wolman Disease

No

Yes

HISTIOCYTIC DISORDERS

 

 

Familial erythrophagocytic lymphohistiocytosis

No

Yes

Hemophagocytosis

No

Yes

Histiocytosis X

No

Yes

Langerhans ‘Cell Histiocytosis

No

Yes

CONGENITAL (INHERITED) IMMUNE SYSTEM DISORDER

 

 

Absence of   T or  B cells

No

Yes

Ataxia Teleangiectasia

No

Yes

Bare Lymphocyte Syndrome

No

Yes

Common Variable Immunoficiency

No

Yes

DiGeorge Syndrome

No

Yes

Kostmann  Sindrome

No

Yes

LeuKocyte Adhesion Deficiency

No

Yes

Omenn’s Syndrom

No

Yes

Severe Combined Immunodeficiency (SCID)

No

Yes

SCID with Adenosine Deaminase Deficiency

No

Yes

Wiskott-Aldrich Syndrome

No

Yes

X Linked Lymphoproliferative Disorder

No

Yes

OTHER INHERITED DISORDER

 

 

Ceroid Lipofuscinosis Congenital

No

Yes

Porphyria Erythropoietic

No

Yes

Glanzmann Thrombasthenia

No

Yes

Lesch-Nyhan Syndrome

No

Yes

Osteopetrosis

No

Yes

Tay Sachs Disease

No

Yes

Sandhoff Disease

No

Yes

INHERITED PLATELET ABNORMALITIES

 

 

Amegakaryocytosis

No

Yes

Congenital Thrombocytopenia

No

Yes

AUTOIMMUNE DISEASES

 

 

Evans Syndrome

Yes

Yes


Legend:
Yes
: Using cord blood cell transplant can cure the disease
No: Transplant cannot be made
Yes*: Cord Blood cell transplant used only in cases of some sub-types of diagnosys. Alogen transplant is prefered if available
Yes& : in case of diagnosys sub-types with great risks, alogen transplant is prefered if available
Yes#: Using alogen transplant in case of adult diseases has limited results. There are no results regarding autolog transplantation yet. Alogen transplant is prefered
Da@: the transplant is usable but there is no cure only extending life expectation
Da! : in case of failure of bone marrow transplant, an alogen one is prefered

Adjuvant Therapy

Disease

Autolog Transplant

Alogen Transplant

Central Nervous System Tumors

Yes

Yes

(experiment)

Ewing’s Sarcom

Yes

Yes (experiment)

Retinoblastoma

Yes

Yes

Neuroblastoma

Yes

Yes

Testicular Cancer

Yes

Yes

 

Clinical trial therapies

 

Malignant tumors (adjuvant therapy)

Breast cancer
Renal cell carcinoma
Ovarian cancer
Small cell lung cancer

 

 

Central Nervous System Diseases

Multiple sclerosis

 

Other Inherited Immune system disorder

Boala Gunther (porfiria eritropoetica)
Hermansky –Pudlak Sindrom 
Pearson Sindrom
Shwachman-Diamond Sindrom
Sistemic Mastocitosis

Gene Therapy

Glanzmann  Thrombasthenia

Severe combined Immunodeficiency

 

Experimental therapy

 

 

Autoimmune diseases

Juvenile Rheumathoid Arthritis
Rheumatoid Polyarthritis
Crohn’s Disease
Systemic lupus erythematosus
Juvenile Dermatomyositis
Sclerodermia

 

 

Terapie reparatorie neuronala

Central Nervous System Diseases

  • Lateral amyotrophic sclerosis
  • Alzheimer Disease
  • Huntington Disease
  • Parkinson Disease

Post cerebral stroke
Spinal cord acute trauma

 

 

Organic Regenerative Therapy

Renal regenerative therapy by asociated renal transplant and stem cell transplant

Liver – Regenerative Therapy
Heart – Acute Myocardial Infarct

Muscular Diseases

Gene Therapy

Fanconi Anemia
Metabolic Congenital Disease
Parkinson Disease

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