Hematopoietic Stem Cell Transplantation

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Hematopoietic Stem Cell Transplantation

Authors
Karam Khaddour1; Caroline K. Hana2; Prerna Mewawalla3.
Affiliations
1 Rosalind Franklin University of Medicine
2 University of Miami
3 Allegheny Health Network Cancer Inst
Last Update: June 27, 2022.
Continuing Education Activity

Bone marrow transplant, also known as hematopoietic stem cell transplant (HPSCT) involves the administration of
healthy hematopoietic stem cells to patients with dysfunctional or depleted bone marrow. This helps to augment bone
marrow function and, depending on the disease being treated, leads to either destruction of malignant tumor cells or to
generation of functional cells that can replace the dysfunctional ones, as is the case of immune-deficiency syndromes,
hemoglobinopathies, and other diseases. This activity reviews the indications, contraindications, and complications
associated with hematopoietic stem cell transplants and highlights the role of the interprofessional team in the
management of patients requiring this treatment.
Objectives:
Describe the indications for hematopoietic stem cell transplants.
Review the contraindications to hematopoietic stem cell transplants.
Outline the potential complications of hematopoietic stem cell transplants.
Describe the need for a well-integrated, interprofessional team approach to improve care for patients undergoing
hematopoietic stem cell transplants.
Access free multiple choice questions on this topic.
Introduction
Bone marrow transplant (hematopoietic stem cell transplant) (HPSCT) involves the administration of healthy
hematopoietic stem cells in patients with dysfunctional or depleted bone marrow. This helps to augment bone marrow
function and allows, depending on the disease being treated, to either destroy tumor cells with malignancy or to
generate functional cells that can replace the dysfunctional ones in cases like immune deficiency syndromes,
hemoglobinopathies, and other diseases.
History and Evolution
Hematopoietic stem cell transplantation (HSCT) was first explored in humans in the 1950s and was based on
observational studies in mice models which showed that infusion of healthy bone marrow components into a
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myelosuppressed bone marrow could induce recovery of its function in the recipient.[1] These animal-based studies
soon found their clinical application into humans when the first successful bone marrow transplant was performed in
monozygotic twins in New York in 1957 (syngeneic transplant) in a patient with acute leukemia.[2] As a result, the
physician Dr. Thomas who performed the procedure continued his research on the development of bone marrow
transplantation and later received the Nobel Prize of physiology and medicine in appreciation of his work. The first
successful allogeneic bone marrow transplant was reported in Minnesota in 1968 for a pediatric patient with severe,
combined immunodeficiency syndrome.[3] Since then, allogeneic and autologous stem cell transplant has increased in
the United States and worldwide. The Center for International Blood and Marrow Transplant Research (CIBMTR)
reported over 8000 allogenic transplants performed in the United States in 2016 with a higher number of autologous
transplants with a steady and higher increase of autologous compared to allogenic.[4]
Definitions
Major Histocompatibility Complex (MHC)
The group of genes on the short arm of chromosome 6 (p6) that encodes human leukocyte antigens (HLA) which are
considered being highly polymorphic leading to a large difference in the resultant expressed proteins on human cells.
They are divided into MHC I and MHC II
Human Leukocyte Antigens (HLA)
These are the proteins expressed on the cellular surface and play an important role in alloimmunity. HLA can be
divided into (HLA-A, B, and C) which are encoded by class I MHC and are expressed on all cell types and present
peptides derived from the cytoplasm and are recognized by CD8+ T cells. The other HLA type is classified as (HLA-
DP, DQ, and DR) which are encoded by MHC II and can be found on antigen-presenting cells (APCs) and this class is
recognized by CD4+ T cells.
Syngeneic Bone Marrow Transplantation
The donor and the recipient are identical twins. The advantages include no graft versus host disease (GVHD) and no
graft failure. However, only a tiny number of transplant patients will have the ability to have an identical twin for
transplantation.
Autologous Bone Marrow Transplantation
The bone marrow products are collected from the patient and are reinfused after purification methods. The advantages
include no GVHD. The disadvantage is that the bone marrow products may contain abnormal cells that can cause
relapse in the case of malignancy hence; theoretically, this method cannot be used in all cases of abnormal bone
marrow diseases.
Allogenic Transplantation
The donor is an HLA matched family member, unrelated matched donor or mismatched family donors (haploidentical).
Engraftment
The process of which infused transplanted hematopoietic stem cells produce mature progeny in the peripheral
circulation
Preparative Regimen
This is a regimen that comprises high-dose chemotherapy and/or total body irradiation (TBI) which are administered to
the recipient prior to stem cell infusion to eliminate the largest number of malignant cells and to allow for
immunosuppression in the recipient so that engraftment can occur.
Indications
Malignant Disease
Multiple Myeloma
Autologous stem cell transplant accounts for most hematopoietic stem cell transplants according to CIBMTR in 2016
in the United States. Studies have shown increased overall survival and progression free survival in patients younger
than 65 years old when consolidation therapy with melphalan is initiated followed by autologous stem cell
transplantation and lenalidomide maintenance therapy.[5] The study showed a favorable outcome of high-dose
melphalan plus stem-cell transplantation when compared with consolidation therapy with melphalan, prednisone,
lenalidomide (MPR). It also showed a better outcome in patients who received a maintenance therapy with
lenalidomide.
Hodgkin and Non-Hodgkin Lymphoma
Studies have shown that chemotherapy followed by autologous stem cell transplantation in cases of recurrent
lymphomas (HL and NHL) that do not respond to initial conventional chemotherapy have better outcomes. A
randomized controlled trial by Schmitz N et al. showed a better 3-year outcome of high-dose chemotherapy with
autologous stem cell transplant compared to aggressive conventional chemotherapy in relapsed chemosensitive
Hodgkin lymphoma. However, the overall survival was not significantly different between the two groups.[6] The
number of hematopoietic stem cell transplant recipient comes second after multiple myeloma according to CIBMTR.
Acute Myeloid Leukemia
Allogenic stem cell transplant has shown to improve outcome in patients with AML who fail primary induction therapy
and do not achieve compete response and may prolong overall survival.[7] The study recommended that early HLA
typing for patients with AML can help if they fail induction therapy and are considered for bone marrow transplant.
Acute Lymphocytic Leukemia
Allogenic stem cell transplant is indicated in refractory and resistant cases when induction therapy fails for a second
time in inducing remission. Some studies suggest an increased benefit of allogenic hematopoietic stem cell transplant
in patients with high risk ALL including patients with Philadelphia chromosome and those with t(4, 11).[8]
Myelodysplastic Syndrome
Allogenic stem cell transplant is considered being curative in cases of disease progression and is only indicated in
intermediate- or high-risk patients with MDS.
Chronic Myeloid Leukemia/Chronic Lymphocytic Leukemia
Recipients with these two diseases come at the bottom of the list of patients who received allogeneic stem cell
transplant in 2016. Hematopoietic stem cell transplantation has shown high cure rates but with available treatments like
tyrosine kinase inhibitors and high success rates with low adverse risk profile, HSCT is reserved for patients with the
refractory disease to first-line agents in CML.
Myelofibrosis, Essential Thrombocytosis, and Polycythemia Vera
Allogenic stem cell transplant has shown to improve outcomes in patients with myelofibrosis and those who had a
diagnosis of myelofibrosis that was preceded by essential thrombocytosis and polycythemia vera.[9]
Solid Tumors
Autologous stem cell transplant is considered the standard of care in patients with germ cell tumor (testicular tumors)
that are refractory to chemotherapy (after the third recurrence with chemotherapy).[10] HSCT has also been studied in
medulloblastoma, metastatic breast cancer, and other solid tumors.
Non-Malignant Diseases
Aplastic Anemia
Systematic and retrospective studies have suggested an improved outcome with hematopoietic stem cell transplant in
acquired aplastic anemia when compared with conventional immunosuppressive therapy.[11] Allogenic stem cell
transplant has shown better outcomes when it was collected from bone marrow compared to peripheral blood in a study
that involved 1886 patients with acquired aplastic anemia.[12] Patients with aplastic anemia need preparative regimen
given they still can develop immune rejection to the graft.
Severe Combined Immune Deficiency Syndrome (SCID)
Large retrospective studies have shown increased overall survival in infants with SCID when they received the
transplant early at birth before the onset of infections.[13]
Thalassemia
Allogenic stems transplant from a matched sibling donor is considered an option to treat Thalassemia and has shown
15-year survival reaching 80%. However, recent retrospective data showed similar overall survival compared with
conventional treatment that consists of multiple transfusions in the case of thalassemia.[14]
Sickle Cell Anemia
Allogenic stem cell transplant is recommended for the treatment of sickle cell disease.[15]
Other Nonmalignant Diseases
Stem cell transplant has been used in the treatment of chronic granulomatous disease, leukocyte adhesion deficiency,
Chediak-Higashi syndrome, Kostman syndrome, Fanconi anemia, Blackfan-Diamond anemia, and enzymatic
disorders. Moreover, the role of stem cell transplant is being explored in autoimmune diseases including systemic
sclerosis, systemic lupus erythematosus and has already shown promising results in cases like relapsing-remitting
multiple sclerosis.[16]
Contraindications
There are no absolute contraindications for hematopoietic stem cell transplant.
Equipment
Special equipment exists for the collection, preservation, and administration of stem cell products.
Personnel
An interprofessional team approach is a mainstay of ensuring the high-quality collection and infusion of stem cell
products.
Preparation
Preparation includes:
Preparative regimen
Collection of hematopoietic stem cells
Instant infusion or cryopreservation followed by infusion
Technique
Mechanism of Action
The mechanism of action of stem cell transplant against malignancy in leukemia is based on the effect of the graft and
donor immunity against malignant cells in recipients. These findings were demonstrated in a study that involved over
2000 patients with different leukemia. These patients received stem cell transplantation and showed that the lowest rate
of relapses was in patients who received non-T-cell-depleted bone marrow cells and in those who developed GVHD
compared to patients who received T-cell-depleted stem cells, those who did not develop GVHD, and patients who
received syngeneic grafts. These findings support the notion that donor cellular immunity plays a central role in the
engraftment's efficacy against tumor cells.[17]
The mechanism of action in autoimmune diseases is believed to be secondary to the increase in T-cell regulatory
function which promotes immune tolerance. However, more studies are still needed to determine the exact
pathophysiology.
In hemoglobinopathies, the transplanted stem cells produce functional cells after engraftment that replaces the diseased
cells.
Administration
HLA Typing
HLA typing is an important step to determine the best donor suitable for stem cell collection. In theory, matched,
related donors are the best candidates, followed by matched unrelated donors, cord blood, and then haploidentical
donors. HLA typing is analyzed at either an intermediate-resolution level, which entails the detection of a small
number of matched alleles between the donor serum and the recipient, or at a high-resolution level to determine the
specific number of polymorphic alleles at a higher level. PCR and next-generation sequencing are used for HLA
typing, and the results are reported as a score correlating with a match of two alleles for a specific HLA type. Different
institutions use a different number of HLA subtypes for eligibility of donors but according to studies that showed
matching for HLA-A, B, C, and DRB1 at a high-resolution level were associated with improved survival and
outcomes.[18] Recommendations about donor HLA assessment and matching have been proposed by the Blood and
Marrow Transplant Clinical Trials Network (BM CTN).[19]
The process may vary depending on the source of the stem cell site collection, whether it is bone marrow, peripheral
blood, or cord blood. Moreover, there is a slight difference based on whether it is autologous, allogeneic, or syngeneic.
For example, the procedure consists of initial mobilization of stem cells, in which peripheral blood stem cells are
collected given the low number and the need for high levels of progeny cells, and then this is followed by preparative
regimen and finally, infusion.
Mobilization and collection involved the use of medication to increase the number of stem cells in the peripheral blood
given that there are not enough stem cells in the peripheral blood. The agents used include granulocyte colony-
stimulating factors (G-CSF) or chemokine receptor 4 (CXCR4) blockers like plerixafor. G-CSF is believed to enhance
neutrophils to release serine proteases which lead to a break of vascular adhesion molecules and the release of
hematopoietic stem cells from the bone marrow. Plerixafor blocks the binding of stromal cell-derived factor-1-alpha
(SDF-1) to (CXCR4) which leads to the mobilization of stem cells to the peripheral blood.[20] CD34+ is considered
the marker for progenitor hematopoietic stem cells in the peripheral blood, and usually, a dose of 2 to 10 x 10/kg
CD34+ cells/kg is needed for proper engraftment. Chemotherapy can be used in some instances for mobilization of
hematopoietic stem cells; this process is termed chemoembolization.
The usual site of bone marrow collection is the anterior or posterior iliac crest. The procedure can be performed under
local or general anesthesia. Complications include pain, fever, and serious iatrogenic complications can occur in less
than 1% of cases. Multiple aspirations are done with each aspirate containing 15 mL. The goal is to collect up to 1 to
1.5 L of bone marrow product from the aspirations. The dose of nucleated cells from bone marrow should range
between 2 to 4 x 10 cells/kg as studies showed that overall survival and long-term engraftment is strongly influenced
by cell dose in allogeneic hematopoietic stem cell transplantation.[21]
The preparative regimen consists of administration of chemotherapy with or without total body irradiation for the
eradication of malignant cells and induction of immune tolerance for the transfused cells to engraft properly. This
process is not only limited to patients with malignancies but also extends to cases like aplastic anemia and
hemoglobinopathies given that these patients have an intact immune system that could cause graft failure if there is no
conditioning.
The preparative regimen is divided into myeloablative conditioning and reduced intensity conditioning. The
preparative regimen depends on the disease being treated, existing comorbidities, and the source of the harvested
hematopoietic stem cells. The preparative regimen consists of chemotherapy, total body irradiation, or both. There are
different combination regimens used in the preparative period, and the choice of the regimen depends on the disease
being treated, existing comorbidities, and previous exposure to radiation.
In the special case of SCID, there is no need for preparative regimen in patients receiving from HLA-matched siblings
given that there are no abnormal cells that are needed to be eliminated and because immunosuppression caused by
SCID can prevent graft rejection. Reduced-intensity conditioning is preferred in patients with prior radiotherapy, older
age, the presence of comorbidities, and history extensive chemotherapy before BMT.[22] The advantages of using
reduced-intensity conditioning include less need for transfusion due to the transient post-transplant pancytopenia and
less damage to the liver in cases of chemotherapy and lung due to radiation.[23] However, the relapse rates are higher,
but these regimens are more tolerated with a better safety profile in a specific patient population. Most of the
chemotherapies used in preparative regimens consist of either potent immunosuppressive agents (high doses of
cyclophosphamide 60 mg/kg IV), alkylating agents especially busulfan 130 mg/m2 IV, nucleoside analogs (fludrabine
40 mg/m2) and other agents like melphalan, antithymocyte globulin, rituximab, gemcitabine, and many others.
Total body irradiation (TBI) is performed using fractionated doses because it has shown less pulmonary toxicity when
compared with one dose regimen.[24] The administration of the preparative regimen should immediately precede the
bone marrow transplantation, and as a general rule, the effect of the regimen should produce bone marrow suppression
within 1 to 3 weeks of administration.
Reinfusion of either fresh or cryopreserved stem cells can occur in an ambulatory setting and takes up to 2 hours.
Before the infusion begins, quality measures are performed to ensure the number of CD34+ cells is sufficient.
Advantages and Disadvantages of Different Hematopoietic Stem Cells
The advantages of peripheral blood stem cells transplant (PBSCTs) include more rapid engraftment rate compared to
bone marrow where it takes about 2 weeks of recovery in the former and is delayed for 5 days more in the latter,
[25] but the use of post-transplant immunosuppressive regimen to prevent GVHD can prolong the increase in bone
marrow products. Moreover, the rate of acute GVHD appears to be similar when compared with bone marrow
transplantation in HLA- identical matched related donors.[25] However, chronic GVHD appears to be encountered
more after peripheral blood stem cell transplant which could lead to more complications.[26] In the study by Anasetti
et al., the primary endpoint was the difference in the 2-year overall survival seemed to be non-significant in the two
groups. However, secondary end points showed more stable grafts with decreased graft failure in the group which
received peripheral blood stem cell transplant but also this group had a higher incidence of chronic GVHD.[26] Other
similar studies comparing both bone marrow transplant and peripheral blood SCTs concluded that the psychological
burden due to chronic GVHD and the 5-year ability to restore normal activities including going back to work were
better in the bone marrow transplanted group.[27]
The advantages of cord blood transplant include the rapid collection and administration which serves in treating urgent
conditions, less frequency of infections, lower rates of GVHD with the same rate of GVT, less need for a stringent
identical HLA. The disadvantages include delayed engraftment with a higher possibility of graft rejection and higher
rates of disease relapses. The cord blood transplant is most used in patients without matched related or unrelated
donors. A major study by Locatelli et al. demonstrated the utility of cord blood transplant in patients with thalassemia
major and sickle cell anemia and showed similar 6-year overall survival in the CBT and BMT groups.[28] The most
important factors that affect the outcome of CBT are the total nucleated cell dose and HLA matching with a
recommended minimum dose of total nucleated cells of 2 x 10*7 cells/kg for successful engraftment. Theoretically,
strict HLA matching is not required in the case of cord blood transplant given it is devoid of mature T cells but studies
have shown better outcomes when matching recipients at HLA-A, HLA-B, HLA-C, and HLA-DRB1.[29] Given that a
single blood cord unit might not contain the required amount of nucleated cells an approach using double cord
transplant is used to overcome this problem. However, only one cord blood transplant product will predominate within
3 months of infusion. Further, randomized controlled trials failed to show a significant difference in terms of outcome
benefits or risks between double cord blood and a single cord blood transplant.[30][31]
Haploidentical stem cell transplantation refers to the administration of bone marrow products from a first degree
related haplotype-mismatched donor.[32] This helps in non-white patients like African American, Hispanics, and
patients from countries where there is no wide access to resources as they have fewer chances of having a matched
unrelated donor.[33] The advantages include lower cost and rapid availability of the hematopoietic cell products.
However, the disadvantages include hyperacute GVHD which increases mortality and graft rejection.[34] This has
been overcome by depletion of T cells responsible for the reaction mentioned above, but this also leads to delayed
immune recovery and decreased graft versus tumor effect. Recently strategies including selective depletion of subsets
of T cells including alpha-beta have shown improved outcomes when compared to conventional ex vivo depletion of
large T-cell populations.[35]
Complications
Complications after bone marrow transplant can be divided into acute and chronic. Many factors can affect the
occurrence of these adverse events including the age of the patient, baseline performance status, the source of stem cell
transplant the type and intensity of the preparative regimen. Acute complications can occur in the first 90 days and
include myelosuppression with neutropenia, anemia, thrombocytopenia, sinusoidal obstruction syndrome (SOS),
mucositis, acute graft versus host disease, gram-positive/gram-negative infections, HSV, CMV, Candida, and
Aspergillus. Chronic complications include chronic GVHD, infection with encapsulated bacteria and VZV.
Levofloxacin is usually given by mouth or intravenously (IV) at day 1 post-transplant and is continued until absolute
neutrophil count (ANC) is more than 1000 cells/uL or until the discontinuation of prednisone in cases of GVHD.
[36] PCP prophylaxis is warranted given the immunosuppression following hematopoietic stem cell transplant.
[37] Trimethoprim-sulfamethoxazole (TMP-SMX) is usually used, and several dosing regimens have been proposed.
TMP-SMX is given 2 days per week until the patient is off immunosuppression.[38] Antifungal infection prophylaxis
with fluconazole is recommended for 1 month following transplant as it has shown to decrease the incidence of fungal
infections and no difference was seen when fluconazole was compared to voriconazole.[39][40] However,
voriconazole is used in patients with a high-risk profile of developing severe forms of antifungal infection. Prevention
against HSV and VZV is achieved with acyclovir that is continued for 1 month for the prevention against HSV and for
1 year for preventions against VZV. [41] Prophylaxis against CMV is only recommended in patients who test positive
for CMV by PCR, and the treatment of choice is ganciclovir.
One unique syndrome encountered with cord stem cell transplant is cord colitis which involves diarrhea in recipients of
cord blood and is believed to be secondary to Bradyrhizobium enterica[42] which usually responds to a course of
metronidazole or levofloxacin.
Sinusoidal Obstruction Syndrome (SOS)
Also known as veno occlusive disease (VOD) is the result of chemotherapy during preparative regimen and occurs
within 6 weeks of hematopoietic stem cell transplant. This syndrome consists of tender hepatomegaly, jaundice due to
hyperbilirubinemia, ascites, and weight gain due to fluid retention. The incidence is reported to be 13.6% in an analysis
study assessing the existing literature on the incidence of the disease.[43] The pathophysiology consists of endothelial
damage to the hepatic sinusoids leading to obstruction and necrosis of the centrilobular liver.[44] The destruction of the
sinusoids leads to hepatic failure and hepatorenal syndrome which are responsible for the related mortality. The agents
most commonly implicated in causing this syndrome are oral busulfan and cyclophosphamide. The use of IV busulfan
has shown to decrease the occurrence of SOS.[45] The diagnosis is clinical and is based on hyperbilirubinemia greater
than 2 mg/dL with the other clinical findings of tender hepatomegaly and fluid retention. Treatment consists of
ursodeoxycholic acid (UDCA) which has shown to significantly decrease the occurrence of SOS when given pre and
post-transplant.[46] Another medication, defibrotide, has shown efficacy in the treatment of SOS when it occurs.[47]
[48]
Idiopathic Pneumonia Syndrome (IPS)
This usually happens in the first 90 days post-transplant. The incidence is low and is related to direct chemotoxicity
due to preparative regimen. Treatment with steroids is usually used although no randomized controlled clinical trials
have been done to support their efficacy. Recently, etanercept has been studied in a randomized control trial in patients
who develop this syndrome. The study assessed the addition of soluble TNF inhibitors to steroids in the treatment and
has not shown added efficacy with combination therapy.[49]
Graft Rejection
The process of which there is a loss of bone marrow function after reconstitution following infusion of hematopoietic
stem cells or if there is no gain of function after infusion and is termed graft failure or rejection. The incidence of
failure is highest when there is a high HLA disparity that usually occurs in the case of cord blood and haploidentical
donors and is lowest with autologous and matched donor siblings. Factors responsible for graft failure include but are
not limited to functional residual host immune response to the donor cells, a low number of infused cells, in vitro
damage during collection and cryopreservation, inadequate preparative regimen and infections.
Chimerism refers to the presence of a cell population from a person in the blood of a different person. Checking for
chimerism is an important step in ensuring engraftment and success of the transplantation. The physician does this by
checking the expression of CD33 which indicates the presence of granulocytes and CD3 which indicate the presence
of T cells and confirming that most of the present cells are from the donor. The importance of effective chimerism has
been demonstrated in many studies that showed decreased relapse rates and increased survival in allogeneic
transplantation.[50]
Graft Versus Host Disease
The graft versus host disease (GVHD) consists of the reaction between T-cells from the donor in allogeneic transplant
and recipient’s HLA polymorphic epitopes that leads to a constellation of symptoms and manifestations. GVHD can be
categorized into acute and chronic which are both sub-categorized into classic and late onset, classic, and chronic
overlap respectively.[51] Acute GVHD usually develops within three months. However, it can develop after 3 months
and is termed delayed acute GVHD. Prophylaxis is usually achieved with calcineurin inhibitors, methotrexate, and
anti-thymocyte globulins. Severity is estimated based on Glucksberg scale which classifies acute GVHD from grade I
to VI, and treatment with either high dose prednisone or methylprednisolone is indicated in higher grades.[52]
Chronic GVHD occurs after three months and is represented by the involvement of multiple organs in a similar fashion
to collagen vascular diseases. Grading has been developed by NIH (Global Grading System) to assess the severity of
GVHD which determines the treatment modality and predicts survival [53]. Treatment is similar to that of acute
GVHD, but the duration is usually over 2two years.[54]
Toxicity
Chemotherapy and radiation of preparative regimen along with post-transplant immunosuppression can induce severe
pancytopenia in the first week following infusion of hematopoietic stem cells which can lead to life-threatening
infection. This depends on the type and the dose of chemotherapy administered and factors related to the recipients.
Chemotherapy causes a destruction of healthy, normal bone marrow products including neutrophils, macrophages,
monocytes, and lymphocytes. Also, mucositis toxicity due to chemotherapy disrupts the barriers protecting against
infectious agents, and use of indwelling intravenous catheters provides another mean of the entrance of infectious
agents. Vaccination is recommended for the following agents according to the guidelines: pneumococcal conjugate
(PCV), TDaP, Haemophilus influenzae, meningococcal conjugate, onactivated polio, recombinant hepatitis B,
inactivated influenza, and MMR.[36] Several regimens of prophylaxis have been proposed to prevent infections
depending on the risk stratification of patients (low-risk, high-risk, treatment of ongoing GVHD.
Many risk scoring tools exist for the evaluation of recipients of hematopoietic stem cell transplant and stratification of
risks so that proper preparation and treatment can be established to minimize the risks and toxicities before, during, and
after transplantation. Some of the most commonly utilized scores in clinical practice are the European Group for Blood
and Marrow Transplantation (EBMT) risk score,[55] hematopoietic cell transplantation-comorbidity index/age,
[56] and Armand disease risk index (DRI).[57]
Enhancing Healthcare Team Outcomes

Hematopoietic stem cell transplant use in clinical practice has been expanding in the last decade, and many clinical
trials are still ongoing to assess its efficacy in different medical conditions.
Review Questions
Access free multiple choice questions on this topic.
Comment on this article.
References
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StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan-.

Karam Khaddour1; Caroline K. Hana2; Prerna Mewawalla3.

Last Update: June 27, 2022.

Continuing Education Activity

Describe the indications for hematopoietic stem cell transplants.

Review the contraindications to hematopoietic stem cell transplants.

Outline the potential complications of hematopoietic stem cell transplants.

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History and Evolution

Major Histocompatibility Complex (MHC)

Human Leukocyte Antigens (HLA)

Syngeneic Bone Marrow Transplantation

Autologous Bone Marrow Transplantation

Hodgkin and Non-Hodgkin Lymphoma

Acute Myeloid Leukemia

Acute Lymphocytic Leukemia

Chronic Myeloid Leukemia/Chronic Lymphocytic Leukemia

Myelofibrosis, Essential Thrombocytosis, and Polycythemia Vera

Severe Combined Immune Deficiency Syndrome (SCID)

Sickle Cell Anemia

Other Nonmalignant Diseases

Collection of hematopoietic stem cells

Instant infusion or cryopreservation followed by infusion

Advantages and Disadvantages of Different Hematopoietic Stem Cells

Sinusoidal Obstruction Syndrome (SOS)

Idiopathic Pneumonia Syndrome (IPS)

Graft Versus Host Disease

Enhancing Healthcare Team Outcomes

Comment on this article.

Cowen EW, Dinndorf P, Farrell A, Hartzman R, Henslee-Downey J, Jacobsohn D, McDonald G, Mittleman B,

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