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High‐dose chemotherapy followed by autologous hematopoietic stem cell transplantation for nonrhabdomyosarcoma soft tissue sarcomas

We reviewed the evidence about the effect of high‐dose chemotherapy (medicines to kill the cancer) followed by autologous hematopoietic stem cell transplantation compared to standard‐dose chemotherapy on overall survival (time from cancer diagnosis, or treatment, to death from any cause) in people with nonrhabdomyosarcoma soft tissue sarcomas. We found one randomized controlled trial (RCT; a clinical study where people are randomly put into one of two or more treatment groups) comparing both treatments.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2017

Mycophenolate mofetil versus methotrexate for prevention of graft‐versus‐host disease following allogeneic hematopoietic stem cell transplantation

Allogeneic hematopoietic stem cell transplantation is a procedure in which a portion of a healthy donor's stem cells (cells that can develop into various types of blood cells) or bone marrow is obtained and prepared for intravenous infusion. Hematopoietic stem cells are taken from a healthy donor and transplanted into the patient (recipient). People undergoing allogeneic hematopoietic stem cell transplantation are at risk of developing graft‐versus‐host disease (GVHD). GVHD results when the transplanted cells from the donor (graft) attack the recipient's (host) body cells because they perceive the recipient's body as foreign. Mycophenolate mofetil and methotrexate are two drugs often used to suppress the human body's reaction against the graft (immune response) and prevent GVHD. We conducted a systematic review of three randomized controlled trials (RCTs, which are clinical studies where people are randomly put into one of two or more treatment groups) that compared mycophenolate mofetil versus methotrexate for use in preventing GVHD among 174 participants. We searched for the relevant studies in March 2014.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2014

Extracorporeal photopheresis treatment for chronic graft‐versus‐host disease after haematopoietic stem cell transplantation in paediatric patients

Chronic graft‐versus‐host disease is a common complication after haematopoietic stem cell transplantation (HSCT; transplant of blood‐forming stem cells). Immune cells (white blood cells) from the donor recognise the patient's cells as foreign ('non‐self'). Therefore, the transplanted immune cells attack the cells of the patient. The main affected organs are skin, liver and gut, among others. These immune reactions may cause acute inflammation (sudden swelling) followed by chronic (long‐term) changes of organs (e.g. fibrosis; scarring of the lungs). First‐line therapy usually consists of immunosuppressive drugs (which reduce the strength of the body's immune system) in the form of corticosteroids in combination with other immunosuppressive agents in refractory cases (where the disease is resistant to treatment). These drugs are supposed to suppress the immune‐mediated attack of the patient's cells. Limited effectiveness and severe side effects of these drugs have led to the application of several alternative approaches.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2016

Extracorporeal photopheresis for acute graft‐versus‐host disease after haematopoietic stem cell transplantation in paediatric patients

Acute graft‐versus‐host disease is a common complication after haematopoietic stem cell transplantation (HSCT; transplant of blood‐forming stem cells). Immune cells (white blood cells) from the donor recognise the patient's cells as foreign ('non‐self'). Therefore, the transplanted immune cells attack the cells of the patient. The main affected organs are skin, liver and gut among other organ tissues. These immune reactions may cause acute inflammation (sudden swelling) followed by chronic (long‐term) changes of the organs (e.g. fibrosis; scarring of the lungs). First‐line therapy usually consists of immunosuppressive drugs (which reduce the strength of the body's immune system) such as corticosteroids in combination with other immunosuppressive agents in refractory cases (where the disease is resistant to treatment). The use of these immunosuppressive drugs is designed to suppress the immune‐mediated attack of the patient's cells. Limited effectiveness and severe side effects of these immunosuppressive drugs have led to the application of several alternative approaches.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2016

Hematopoietic stem-cell transplantation in the pediatric population

Bibliographic details: Ratko TA, Belinson SE, Brown HM, Noorani HZ, Chopra RD, Marbella A, Samson DJ, Bonnell CJ, Ziegler KM, Aronson N.  Hematopoietic stem-cell transplantation in the pediatric population. Rockville, MD, USA: Agency for Healthcare Research and Quality. Comparative Effectiveness Review; 48. 201222439159

Database of Abstracts of Reviews of Effects (DARE): Quality-assessed Reviews [Internet] - Centre for Reviews and Dissemination (UK).

Version: 2012

Donor versus no donor comparison of hematopoietic cell transplantation for adult acute lymphoblastic leukemia in first complete remission

An area of uncertainty in the care of patients with acute lymphoblastic leukemia (ALL) is the choice of treatment that is given after a complete remission is achieved with induction chemotherapy. Therapeutic alternatives include consolidation chemotherapy, autologous transplant (transplantation of a patient's own stem cells) or allogeneic transplant (transplantation utilizing donor stem cells). Clinical trials have come to different conclusions about the best approach. We conducted a systematic review and meta‐analysis to synthesize available clinical research studies that have examined outcome according to donor vs. no donor status, or genetic randomization. This is a method of analysis for assessing the effect of transplantation in this disease condition. Our analysis supports matched sibling donor allogeneic hematopoeitic cell transplantation as the approach which offers the best long‐term outcomes, specifically providing optimal survival and reduced risk for ALL relapse.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2011

Corticosteroid regimens for treatment of acute and chronic graft versus host disease (GvHD) after allogenic stem cell transplantation

Corticosteroids are commonly used to treat acute and chronic graft‐versus‐host disease (GvHD) but their effect on length and quality of life of patients has not been studied systematically. In this systematic review, we tried to compare the effect of treatment regimens used for GvHD in the absence and presence of corticosteroids, or with different doses of corticosteroids. After searching relevant sources, we located only two studies that met our criteria to be included in the study. Their results are described in detail in the text of the review. In brief, these studies are in favor earlier remission and slightly better outcome in patients but more evidence is needed in this field.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2010

Stem cell transplantation for treating Gaucher disease

Gaucher disease is an inherited disorder caused by a deficiency of the enzyme glucocerebrosidase. This leads to storage of complex lipids in some types of blood cells. Due to these abnormal cells people with Gaucher disease will have pain, fatigue, anemia, jaundice and bone damage. Some forms of Gaucher disease may also cause neurological disorder. The treatment of Gaucher disease at present is mainly by enzyme replacement therapy which is expensive. In some severe cases stem cell transplantation is used to treat people with Gaucher disease. This is a high risk procedure some times leading to death of the individual. We have not found any trials to show the effectiveness and the risks of the procedure in people with Gaucher disease. Given there are no trials included in the review and we have not identified any relevant trials up to February 2012 we do not plan to update this review until new trials are published.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

Transplantation of blood‐forming stem cells for children with sickle cell disease

We reviewed the evidence about the cure rate and risks of hematopoietic stem cell transplantation for people with sickle cell disease.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2016

Corticosteroids for preventing graft‐versus‐host disease after allogeneic myeloablative stem cell transplantation

Some types of blood cancer can be treated by transplanting stem cells from the patient's blood relatives or siblings. Unfortunately, transplanted stem cells (also called the 'graft') can sometimes induce an inflammatory reaction in the patient (or the 'host'). This reaction is called 'graft‐versus‐host disease' (GvHD), and once it occurs it is difficult to treat. GvHD can adversely affect the patient's quality of life and often causes death. Drug therapies have been developed to prevent GvHD. Even so, many patients still suffer this complication. Preventive therapy against GvHD must be optimised. Since corticosteroids are the first‐line treatment used after GvHD occurs, it is a hypothesis that if used in prophylaxis regimens, corticosteroids can decrease the occurrence of GvHD and improve patient survival rates.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2009

Anti‐thymocyte globulins for the prevention of graft‐versus‐host disease in patients receiving allogeneic stem cell transplantation

Allogeneic haematopoietic (blood) stem cell transplantation (HSCT) is a potentially curative therapeutic option for a variety of malignant and some non‐malignant haematological (blood‐related) diseases. For this therapy, blood stem cells are transferred from a healthy person who has compatible tissue markers, so‐called human leukocyte antigen (HLA) markers, to a matched recipient. Even though the donor and the recipient are matched concerning these markers, immune cells that are part of the transferred cells ('the graft') are prone to recognise tissues of the recipient ('the host') as being to some extent incompatible or foreign, which then can induce inflammation ('graft‐versus‐host‐disease' or 'GVHD'). GVHD typically involves the skin, the gastrointestinal tract and the liver. GVHD is divided into acute and chronic forms based on the clinical features and the time of occurrence after transplantation. In order to prevent this potentially life‐threatening condition, reactive immune cells can be depleted in the recipient by administering antibodies which are directed against them. These antibodies are called anti‐thymocyte globulins (ATG) and are derived from animals which were immunised with human thymocytes or T‐cells. Different types of ATG have been used for decades to decrease the occurrence and severity of GVHD but they bear the risk of severe side effects such as increased infection rates or the risk of disease relapse. Also, severe side effects such as allergic reactions or serum sickness with shortness of breath and fever, blood coagulation disturbances or liver failure can harm the patient during the infusion of ATG. So far, no systematic analysis of the advantages and disadvantages of the use of ATG has been done.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2012

Stem cell transplantation of sibling donors compared with specific immunosuppressive therapy for acquired severe aplastic anemia

Acquired severe aplastic anemia is rare. Stem cells from the bone marrow usually replace naturally dying blood cells in the peripheral blood. Severe aplastic anemia is probably caused by an irregular, attacking immune response against these blood producing stem cells within the body. If supplies are not maintained, functional blood cells are lacking and infections, bleeding, and exhaustion will occur. Patients may experience paleness, weakness, fatigue, and shortness of breath. Disease progression is associated with severe infections, which are a major cause of death.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2013

Prevention of occlusion of small veins in the liver after blood‐forming stem cell transplantation

We reviewed evidence about the effects of medications to prevent blockage of small veins in the liver (veno‐occlusive disease or VOD) in people who undergo blood‐forming stem cell transplantation (HSCT).

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

Adding plerixafor to G‐CSF for stem cell mobilisation for autologous transplantation in people with certain cancers of the blood

We reviewed the existing literature regarding the efficacy and safety of additional plerixafor to granulocyte colony‐stimulating factors (G‐CSF) versus G‐CSF only for stem cell mobilisation in people with malignant lymphoma and multiple myeloma, cancers of the blood.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

Transplantation of blood‐forming stem cells for people with ß‐thalassaemia major

We reviewed the evidence about the effect and safety of different types of allogeneic stem cell transplantation in people with severe transfusion‐dependant ß‐thalassaemia major or ß0/+‐thalassaemia variants requiring chronic blood transfusion.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2016

Stem cell transplantation for treating cancer

Stem cell transplantation is used in treating the various forms of blood cancer, for instance leukemia. It is a very strong, high-dose form of chemotherapy that destroys the stem cells in our bone marrow. The body is then immediately provided with healthy stem cells via the blood, which settle in the bone marrow and produce fresh blood cells.

Informed Health Online [Internet] - Institute for Quality and Efficiency in Health Care (IQWiG).

Version: December 30, 2016

Autologous stem cell transplantation for soft tissue sarcoma: Executive summary of final report N05-03D, Version 1.0

The aim of this investigation was to assess the benefit of autologous hematopoietic stem cell transplantation (HSCT) on patient-relevant outcomes in patients with soft tissue sarcomas compared to a procedure without HSCT.

Institute for Quality and Efficiency in Health Care: Executive Summaries [Internet] - Institute for Quality and Efficiency in Health Care (IQWiG).

Version: September 17, 2009

High‐dose chemotherapy and stem cell transplant compared to conventional therapy for children with high‐risk neuroblastoma

Despite the development of new treatment options, the prognosis of high‐risk neuroblastoma patients still remains poor; in more than half of patients the disease returns. Stem cell rescue replaces blood‐forming stem cells that were destroyed by high‐dose chemotherapy in order to recover the bone marrow. It is also known as myeloablative therapy and might improve the survival of these patients. A well‐informed decision on the use of myeloablative therapy in the treatment of children with high‐risk neuroblastoma should be based on high quality evidence of effectiveness for treating tumours and side effects.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

Autologous stem cell transplantation for breast cancer: Executive summary of final report N05-03E, Version 1.0

The aims of this investigation were to assess studies on certain types of autologous stem cell transplantation in breast cancer patients: compared to cytostatic therapy without stem cell support, and compared with each other.

Institute for Quality and Efficiency in Health Care: Executive Summaries [Internet] - Institute for Quality and Efficiency in Health Care (IQWiG).

Version: October 15, 2009

A comparison of the healthy donor's experience of donating their blood stem cells to a patient who is to receive a stem cell transplant as treatment for cancer of their blood (e.g. leukaemia)

Blood stem cells are collected from a donor in two ways: either through a bone marrow harvest (direct retrieval of the stem cells from the donor's hip bones, under general anaesthetic) or a peripheral blood stem cell collection (retrieval of stem cells using a blood cell separator machine, following a course of granulocyte colony stimulating factor (G‐CSF) injections). Both these methods of donation are common. Much research has explored which method of donation gives the best outcome to the patient, however there has not been a lot of research exploring these methods of donation from the donor's perspective. Such research is important if there is the possibility of long‐term adverse events for the donor. For example, the long‐term adverse events of G‐CSF are not known, but there is the suggestion of a correlation between G‐CSF and development of myelodysplastic syndrome (MDS). However, in many instances, donors are given a choice as to which method they would like to use to donate their stem cells. The aim of this review was to compare directly these two methods of blood stem cell donation from the donor's perspective, to understand the experiences of the donor. In this review, each donor was a sibling of the patient to whom they were donating blood stem cells.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2009

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