artwork-3 · 2017. 9. 15. · 1. graft-versus-host disease (gvhd) this condition is triggered by...

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Gloria (right) is thefirst child patient whor e c e i v e d b o n emarrow transplant inHong Kong. Ther a n s p l a n t w a sperformed at the CCCin February, 1991.

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The Lady Pao Children’s Cancer Centre, Prince of Wales Hospital, aÉé~êíãÉåí=çÑ=m~ÉÇá~íêáÅëIqÜÉ=`ÜáåÉëÉ=råáîÉêëáíó=çÑ=eçåÖ=hçåÖ

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History of Pediatric Bone Marrow TransplantThe idea of using bone marrow to treat blood disease

is not new. In the early 1900, patients were given marroworally. Obviously this did not meet with success and interestfaded. After the Second World War, this treatment strategywas re-evaluated due to the development of nuclear weaponand generators.

Early attempts to transplant bone marrow into humansvia blood vessels were met with mixed success. While afew leukemia patients had transient improvement of theirdisease, most transplants were rejected. Other patientsdeveloped a clinical syndrome of skin rash, diarrhea, andjaundice, which was mostly fatal. The enthusiasm waned inthe 60’s.

Around this time, the genetics of graft rejection andtissue (called HLA) matching was described. For the firsttime, physicians were able to match and select the mostsuitable marrow donor for the patient. The first successfulbone marrow transplant of the modern era was performedin 1968, when an infant born without an intact immunesystem received a transplant from a sibling. This patientremains healthy 35 years later. Similar success was soonreported for patients with severe aplastic anemia and acuteleukemia. By 1986, more than 200 transplant centersworldwide were performing 5,000 transplants annually.

Around this time, it was discovered that stem cells(which produces subsequent generations of blood-formingcells) move freely between the bone marrow and the bloodstream. This exodus of stem cells into the circulation is mostevident during the recovery phase after chemotherapy. Itcan be also enhanced by the administration of proteinscalled colony-stimulating factors (G-CSF). By the middle ofthe 1990’s, many transplant centers were using stem cellscollected from peripheral blood instead of, or in addition to,bone marrow. Such a procedure is called peripheral bloodstem cell transplantation.

‘The latest player in the field is placental blood. Likebone marrow, blood collected from the placenta (also knownas umbilical cord blood) is rich in stem cells. In 1988,researchers in France transplanted placental bloodcollected from a newborn child to the baby’s sibling a

child with Fanconi anemia. Since then successful cord bloodtransplants have performed on children with leukemia,severe aplastic anemia, and a number of other blooddisorders. Due to the small number of stem cells in aplacental blood collection, cord blood transplantation iscurrently only a suitable option only for children and,possibly, small adults. Research is underway to determineif cord blood cells can be expanded so that more adultscan benefit from this therapy.

The majority of patients who could have benefited froma transplant do not have a brother or sister with matchingmarrow type. In 1973, the first unrelated marrow donor(matched for HLA tissue type) transplant was performed inNew York. The success of this procedure led to theestablishment of marrow donor registries around the world.In 1986, the National Marrow Donor Program in the US wascreated. Currently there is information on over 7 millionsvolunteer donors worldwide. Similarly over 20 umbilical cordblood banks are collecting and preserving placental bloodfrom newborn infants. There is international cooperation tofacilitate the identification, testing, and procurement of stemcells to be used for transplantation.

Types of Stem Cell TransplantThe bone marrow is a spongy tissue found inside

bones. It contains stem cells, which divide and generatedifferent kinds of blood cells in the circulation. The whiteblood cells fight infection; the red blood cells carry oxygento body tissues; and the platelets control bleeding.

Diseases affecting the stem cells result in bonemarrow malfunctioning: either by producing too manydefective / immature blood cells (e.g. thalassemia andleukemia), or too few blood cells (apalstic anemia). Whenthese conditions are diagnosed, replacement of the patient’smarrow with stem cells from a healthy donor may lead to acure. This type of transplant is called an allogeneic stemcell transplant. A stem cell transplant may also be part of

Ka Wah Chan MD, *MD Anderson CancerCenter

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Overview of Pediatric BoneMarrow Transplantation

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Medical Frontiers

the treatment for patients with solid tumor cancers. In thesesituations, some of the patient’s own stem cell can becollected and frozen. This allows a higher dose ofchemotherapy to be given to kill the tumors, and the patientcan be “rescued” by reinfusion of the previously stored cells.The use of the patient’s own, otherwise normal, marrow orblood stem cells for treatment is called autologous stemcell transplantation.

How does Stem Cell Transplant Work?Prior to transplant, the patient ‘s diseased bone marrow

has to be destroyed. This usually involves the use of acombination of chemotherapy drugs, or by a combinationof chemotherapy and irradiation, given in very high doses.At the same time the patient’s immune system is alsoeliminated to prevent rejection. This phase of treatment iscalled the preparative or conditioning regimen and typicallylasts five to ten days.

The procedure to collect stem cells differs accordingto nature of the cells used for transplantation. Bone marrowis harvested in a hospital operating theatre. The donor isput under general anesthesia and a needle is inserted intothe hipbone. Over several skin puncture sites repeated bonepunctures are performed. A small amount of bone marrowis extracted each time and the total quantity of marrow ispooled, filtered, and transported to the patient’s room forimmediate infusion. If peripheral blood stem cells are to beused, the donor will receive three to five days of G-CSF tomobilize stem cells to move out of the bone marrow into theblood stream. A needle or special tubing is placed in a largevein either in the donor’s arm or the groin. Blood flows into acomputer-control machine called a cell separator, which willremove the stem cells and return the rest of the blood cells

back to the donor.

On the day of transplant, usually one to three daysafter the completion of conditioning, stem cells are passedinto the patient through a catheter, much like a transfusion.Typically the infusion takes 30 minutes to two hours tocomplete.

Success Rate: Is It Worth All the SideEffects?

The question parents most frequently asked is, “Whatis the success rate of the transplant center in treating mychild’s disease?” It should be stressed that success ratequoted by centers is meaningless unless it is properlyinterpreted.

Many factors influence the success of a stem celltransplant. The most important element is the patient’s statusof disease at the time of transplant. Leukemia that is notunder good control by conventional chemotherapy is verydifficult to cure by a stem cell transplant. There is a higherchance of leukemia recurrence in these cases. When thereis progressive disease a transplant will not help and shouldnot be done. The health condition of the patient is also criticalfor the success of the transplant. Active infection, poorfunction of any organ system, and general debilitation areusually associated with a higher complication rate due tothe effect of conditioning. Finally the donor type affects thetransplant outcome. Autologous and matched sibling donortransplants have a lower complication rate whereasmismatched and unrelated donor transplants are generallymore dangerous.

What Complications Should I be Aware of?1. Graft-Versus-Host Disease (GVHD)

This condition is triggered by the T cells of the donor,which are a type of white blood cells that recognize andattack the body cells of another individual (that of therecipient). The larger the differences in the genetic makeupbetween donor and recipient, the higher the likelihood ofGVHD . On the average 20 to 70% of transplant recipientsmay develop this problem, and as expected the incidenceis higher when stem cells from an unrelated or mismatcheddonor are used.

The acute form of GVHD usually appears during thefirst few weeks after engraftment. The earliest sign is oftena skin rash on the patient’s face and hands. It may spreadto other parts of the body into general redness, similar to asunburn, with peeling and blistering of the skin. The stomachand the bowel may also be affected, causing cramping,

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Yuk Wun (Centre) is the 100th child patient who received transplant atthe CCC in January, 1998.


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Advisors: Prof. Fok Tai Fai, Prof. Patrick Yuen Man PanChief Editor: Dr. Shing Ming Kong Executive Editor: Tang Yuet MayTranslator: James Leung, Elsa Au Design & Production: Constance LamEditorial Committee: Wong Fung Yi, Lo Chun Ha, Pau To Fung Chu,

Ip Fan Ching Yee, Sue & lan Leatham, Christine DugganAcknowledgement: Suen Chi Sun Charitable Foundation for their

support in publishing the newsletter.© Little Life Warrior Society All Rights Reserved.

Editorial Board

Medical Frontiers

* Dr. Ka Wah Chan is the professor of Pediatrics Patient Care, MDAnderson Cancer Center. His clinical interests are on bone marrow andstem cell transplantation for children and adolescents with cancer andblood disorders; placental cord blood transplantation.

Blood and marrow transplantation are nowestablished treatment modality in the managementof childhood cancer, blood and immune disorders.Each year over 2,000 children undergo this treatment.It is a lengthy and vigorous procedure. The decisionto proceed with a transplant must be weighed betweenthe chances of success against the short- and long-term side effects of transplantation.

Summary

body defense to function at 100% efficiency. Recovery isdelayed by GVHD and its treatment.

Extra precaution is necessary to prevent infection aftertransplant. Good hand washing and avoiding contact withothers who are ill are of paramount importance. Closesurveillance and prophylactic antibiotics may also be helpfulto manage the reactivation of some viruses dormant in thepatient’s body.

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Kelvin (left) is the 194th child patient who received transplant at theCCC in July, 2003.

nausea, and watery diarrhea. Jaundice (yellow color of theskin and eyes) is a sign the liver is also involved. Dependingon the number of organs affected and their severity, acuteGVHD may be mild and transient or it may be severe andlife threatening.

The chronic form of GVHD develops several monthsafter the transplant. It may evolve from the acute form or itmay present independently. Patients usually experience skinproblems such as a dry itchy rash, color changes, andtightness. Dryness or burning of the mouth and eyes, liverabnormalities and weight loss are also common complaints.Less frequently, patients may suffer from skin scarring andcontractures, loss of hair and nails, and swallowing andbreathing difficulties.

Both acute and chronic GVHD are treated bymedications that suppress the overactive T cells. Both thediagnosis and its treatment weaken the patient’s immunesystem, so there is an increased risk of opportunisticinfections.

2. Infections

Transplant patients are prone to infections during allphases of the procedure. With high dose chemotherapy and/or radiation during the preparative phase, the bone marrowis destroyed and the white blood cell count becomes verylow. Antibody-producing cells are depleted. The skin andthe lining of the mouth and intestine, the body’s other firstline of defense, are also damaged. The patient is almostcertain to have fever in the first two weeks after transplant.Most of these episodes are responsive to antibiotics,suggesting the infections are bacterial in origin. Occasionallyfungi may be the cause of the fever, especially afterprolonged antibiotic treatment.

Even after the white cell count recovers, usually in twoto four weeks’ time, the function of the patient’s immunesystem remained subnormal. The risk of unusual infectionscaused by virus, protozoa, parasite and mycobacteriumremain significant. It takes 6 to 12 months or more for the

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