Current applications

Cryopreservation of stem cells allows them to be used immediately, either by the donor of the cells or in a compatible family member, for the treatment of a range of diseases

01

Curable diseases

There are now over 80 diseases for which stem cells from umbilical cord blood can be used for treatment:

  • in 5 of which, the patient’s own stem cells have already been used, specifically bone marrow diseases and solid tumors; autologous use is generally for diseases that are not present at birth and are acquired during life.

  • transplants were carried out using cells from related and non-related donors for the rest of the diseases, such as leukemia and metabolic diseases that are normally related to a genetic mutation, i.e. present since birth.

Cancers
Acute lymphoblastic leukemia (all)*1)
Myeloid leukemia (cml)
Chronic myeloid leukemia (cml)
Chronic lymphoid leukemia (cll)
Myelomonocytic leukemia
Solid tumours (e.g. neuroblastoma or retinoblastoma)*
Hodgkin disease
Non-Hodgkin lymphonas*
Refractory anaemia
Myelofibrosis
Systemic mastocytosis
Autoimmune lymphoproliferative syndrome
Genetic histiocytosis
Langerhans cell histiocytosis
Hemophagocytic lymphohistiocytosis
Granulomatosis lymphomatosis
Child chromosome monosomy syndrome 7
Hemoglobinopathies
Beta-thalassemia major
Beta-thalassemia intermedia
Alpha-thalassemia intermedia (Hemoglobin H disease)
Alpha-thalassemia major (Hydrops fetalis)
Sickle-cell disease
Metabolic diseases
Adrenoleukodystrophy
Gunther’s disease
Gaucher’s disease
Hunter syndrome (MPS-II)
Hurler syndrome (MPS-I)
Hurler-Scheie syndrome
Maroteaux-Lamy syndrome (MPS-VI)
Sanfilippo syndrome (MPS-III)
Hermansky-Pudlak syndrome
Type II, III mucolipidosis
Alpha mannosidosis
Neimann pick syndrome
Sandhoff syndrome
Tay Sachs disease
Krabbe’s disease
Metachromatic leukodystrophy
Fucosidosis (fucosidase deficiency disease)
Gm1 gangliosidosis
Wolman’s disease
Aspartylglucosaminuria
Morquio syndrome (MPS-IV)
Lesch-Nyhan syndrome< Austin disease (Multiple sulfatase deficiency)
Immunodeficiencies
Severe combined immunodeficiency (SCID), including
– omenn syndrome
– sciD with adenosine deaminase deficiency (ADA-SCID)*2)
– X-linked sciD
Ataxia telangiectasia syndrome
DiGeorge syndrome
Wiskott Aldrich syndrome
X-linked Agammaglobulinemia
Chronic granulomatous disease
IKK gamma deficiency
Hipogammaglobulinemia
X-linked lymphoproliferative disorder
Griscelli syndrome
Nezelof syndrome
Bone Marrow Deficiencies
Aplastic anaemia
Acquired aplastic anaemia*
Fanconi anaemia
Genetic dyserythropoietic anaemia
Blackfan-Diamond anaemia
Genetic sideroblastic anaemia
Hypo lymphoproliferative anaemia
Pure red cell aplasia
Cyclic neutropenia
Autoimmune neutropenia (severe)
Evans syndrome
Nocturnal paroxysmal hemoglobinuria
Glanzmann’s disease (platelet abnormality)
Amegakaryocytic thrombocytopenia
TAR syndrome (thrombocytopenia with absent radius)
Severe neo-natal thrombocytopenia
Juvenile dermatomyositis
Juvenile xanthogranuloma
Pancytopenia
Kostmanns syndrome
Shwachman-Diamond syndrome*
Pearson syndrome
Other diseases
Osteopetrosis

Information about cell dose in transplants.
MORE INFORMATION

The number of stem cells per kilogram needed for treatment purposes varies depending on the disease in question. In this sense, for some diseases this can mean that the number of stem cells collected from the umbilical cord may only allow for the treatment of children weighing up to 25-30 kg, but for other diseases this limitation may not exist. Along with the disease and the patient’s weight, the recommended number of cells also depends on the degree of compatibility between donor and recipient.

Regarding to autologous transplants, there are some diseases that have already been treated using bone marrow and peripheral blood and for which it will also be possible to use umbilical cord blood, although transplants have not yet been carried out.

This means that autologous umbilical cord blood may become useful for a greater number of diseases than those it has been used for until now.


* in these diseases, umbilical cord blood was used in an autologous context (the donor and recipient are the same person). In recent cases, it has been used in an allogenic context (donor and recipient are different people), for example, between siblings.

1) First report of autologous cord blood transplantation in the treatment of a child with leukemia. Hayani A, Lampeter E, Viswanatha D, Morgan D, Salvi SN. Paediatrics. 2007 Jan;119(1):e296-300.

2) in combination with gene therapy
List drawn up based on the scientific article moise KJ “Umbilical Cord Stem Cells” Obstet Gynecol 2005; 106:1393-1407 and the website: www.parentsguidecordblood.com.

01

Probabilities of use

The probability of needing a hematopoietic transplantation increases with age.
According to a scientific study, at 70 years of age it is around 1/200.

apliatuais

The probabilities shown above are based on a scientific article from 2008. This is the latest study on the probabilities of hematopoietic transplantation (umbilical cord blood, bone marrow or peripheral blood) during life, based on annual estimates of hematopoietic transplants performed in the US, on the frequency of diseases that require hematopoietic transplants and on demographic data from the US.

There are other previously published studies that suggested lower probabilities of use. For example, an opinion article published in 1999* states that the probability of having to use umbilical cord blood in an autologous context is 1:20,000 during the first 20 years of life. However, unlike the 2008 scientific article, these studies lack calculations to justify these probabilities.

Stemlab takes the 2008 article as a reference, as it is more up-to-date and provides a justification for the presented numbers.

 

* Annas, G.J. (1999) ”Waste and longing–the legal status of placental blood banking”. N Engl J Med. 340, 21-4.

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Types of use

There are two types of stem cell use: autologous and allogeneic.

Hematopoietic transplants performed in Europe in 2014

tiposaplica

In autologous transplants, your own stem cells are used1. This is the preferred option for diseases that can be treated with your own stem cells, to avoid complications with incompatibility. Autologous use is more common than allogeneic use for hematopoietic transplants with bone marrow and peripheral blood.

In allogeneic use, the patient is treated with stem cells from a compatible person. The donor may or may not be related to the patient. However, the transplant is more successful when both the donor and patient are related.2 It should be mentioned that the likelihood of compatibility between siblings is 25%.

In the case of transplants with stem cells from umbilical cord blood, the number of autologous transplants is still lower than the number of allogeneic transplants, because these diseases usually occur in older patients who did not have the opportunity to cryopreserve cells from their umbilical cord blood.

The use of umbilical cord blood samples from siblings is preferred over unrelated donors.
MORE INFORMATION

Allogeneic umbilical cord blood transplants with samples from a related donor (such as a sibling) increases the success of the transplant (greater chance of survival and lower chance of graft versus host disease).

Using cells from a related donor makes a difference


Transplantes

Source: based on a study of 45 transplant centres, related to 143 transplants performed between 1988 and 1996. Information from Gluckman E, Rocha V, Boyer-Chammard A, et al. Outcome of cord-blood transplantation from related and unrelated donors. N Engl J Med. 1997;337(6):373-381.


(1) In the case of genetic and/or congenital diseases, autologous use of umbilical cord blood may not be recommended due to the risk that the cells may already be affected by the disease. However, for other types of diseases, having autologous cells available avoids the rejection of the transplant, as well as secondary complications associated with allogenic transplants.
(2) I1Gluckman E et al. (2011) Family-directed umbilical cord blood banking. Haematologica. 96(11):1700-7.
Rocha V et al. (2009) Pediatric related and unrelated cord blood transplantation for malignant diseases. Bone Marrow Transplant.44, 653Ð9.

 

References:
Hematopoietic stem cell transplantation in Europe 2014: more than 40 000 transplants annually. Passweg JR et al., Bone Marrow Transplant. 2016 Feb 22. doi: 10.1038/bmt.2016.20