ALTERNATE NAMES

Allogeneic HCT; Allogeneic HSCT; Autologous HCT; Autologous HSCT; Bone Marrow Transplant; Bone Marrow Transplantation; Cord Blood Transplant; HCT; Hematopoietic Progenitor Cell Transplant; Hematopoietic Progenitor Cell Transplantation; HPCT; HSC; HSCT; LT-HSC; Multipotent Progenitor; Peripheral Blood Stem Cell Transplant; Stem Cell Transplant; Stem Cell Transplantation

DESCRIPTION

Hematopoietic stem cell transplantation (HSCT), also known as stem cell transplantation or bone marrow transplantation, involves administering healthy hematopoietic stem cells, which are immature cells found in bone marrow and peripheral blood that can develop into red blood cells, platelets, and white blood cells, to individuals with dysfunctional or depleted bone marrow. Different hematopoietic stem cell sources may be used in HSCT, including peripheral blood stem cells, bone marrow stem cells, and cord blood stem cells.

There are two main types of HSCT:

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  Autologous – the transplanted cells come from the patient; and

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  Allogeneic – the transplanted cells come from a donor.

HSCT does not include immunotherapies (immune effector cell therapies), such as chimeric antigen receptor T cell (CAR-T cell) therapy.

An individual’s immune system remains impaired for up to 12 months after receiving an HSCT and the individual generally remains severely immunocompromised. There is high risk of the underlying condition(s) returning, developing new organ damage, or developing severe infections or diseases. Because of the severe immune compromise, the high risk of complications, and the difficult recovery period during this time, the 12 months after the date of transplantation is considered CAL-level impairment. Note: Complications do not have to occur in order to meet CAL requirements.

HSCT can be used to treat several malignant (cancerous) conditions, such as leukemias or lymphomas, and non-malignant conditions, such as conditions involving dysfunction of the hematological or immune systems, in adults and children. HSCT may also be used to treat conditions such as multiple sclerosis or Hurler’s syndrome.

DIAGNOSTIC TESTING, PHYSICAL FINDINGS, AND ICD-9-CM/ICD-10-CM CODING

Diagnostic testing: Diagnostic testing is completed after the HSCT to monitor engraftment (when transplanted stem cells produce mature cells), detect relapse, identify graft failure, and assess graft-versus-host-disease (GVHD).

An individual may experience side effects and complications related to the transplant or post-transplant treatment. Testing to diagnose these conditions will vary based on the type of condition and the symptoms each individual may experience.

Physical findings: An individual may experience severe complications after receiving an HSCT. These complications usually occur because of the conditioning regimen, also known as a preparative regimen, which includes the use of high-dose chemotherapy or total body irradiation or both to destroy bone marrow cells in order to create space in the bone marrow for the healthy stem cells to engraft and to suppress the immune system. Other complications occur because the transplanted hematopoietic stem cells did not engraft; or once engrafted, treat the recipient’s body as foreign and attack it.

The most common complications that occur after an HSCT are acute or chronic GVHD and pancytopenia. Other complications can include frequent or life-threatening infections or deterioration of organ systems. These complications usually occur in the first 12 months after transplantation.

An individual with pancytopenia may experience signs and symptoms that may include:

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  Anemia (low red blood cells);

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  Leukopenia (low white blood cells);

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  Thrombocytopenia (low platelets);

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  Fatigue;

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  Weakness;

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  Dizziness;

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  Shortness of breath;

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  Easy bruising and bleeding; and

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  Frequent infections.

Acute or chronic GVHD generally affects the skin, gastrointestinal tract, and liver. Signs and symptoms of GVHD may include:

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  Rash;

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  Bullous lesions (large, fluid-filled blisters (bullae) on the skin or mucous membranes);

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  Diarrhea;

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  Abdominal pain;

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  Dry, burning eyes;

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  Dryness or painful sores in the mouth;

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  Nausea;

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  Vomiting;

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  Loss of appetite;

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  Yellowing of the skin and eyes (jaundice);

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  Elevated bilirubin and alkaline phosphatase levels;

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  Severe coagulopathy (impaired ability for blood to clot);

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  Severe hyperammonemia (high levels of ammonia in the blood);

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  Enlarged liver (hepatomegaly);

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  Pale urine; and

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  Pale (acholic) stool.

ICD-9: 279.5; 284.1; 996.85; 999.89; V42.81; V42.82

ICD-10: D61.81; D61.89; D89.810; D89.811; D89.812; T86.0; T86.5

PROGRESSION

HSCT recipient survival rates vary greatly depending on a number of factors, including the underlying condition and patient- and donor-related factors. There has been some improvement in survival due to advances in HSCT methods and supportive care and it is now a potentially curative treatment for some conditions. Despite these improvements, disease relapse and acute and chronic complications continue to affect survival rates.

Complications are common within one year after the transplant. These complications are often caused by the conditioning regimen, delayed immune reconstitution, and GVHD. Those with complications have higher rates of inpatient readmission to the hospital in the 12-months following transplantation. The risk and type of complications vary and are influenced by patient-related factors such as age, performance status, and comorbidities.

TREATMENT

The transplant occurs when the healthy hematopoietic stem cells are administered to the recipient as an intravenous (IV) infusion. Additional IV medications may be provided immediately before the infusion to prevent complications. The day of the infusion is considered the date of transplantation.

The post-transplant hospitalization period typically lasts through engraftment, which usually takes 10-21 days. During this period, treatment generally includes daily blood count monitoring, antibiotic or antiviral prophylaxis, and use of precautions to avoid exposure to germs. Supportive and, occasionally, intensive care may be required to treat complications during the period between conditioning and engraftment.

Because the immune system is severely impaired, the transplant recipient may receive treatment for complications such as infections or immunodeficiency during the 12 months after the HSCT. This treatment may include monitoring for potential relapse of the underlying condition; evaluation and management of chronic GVHD; and evaluation and treatment of late complications, such as cardiovascular disease, respiratory disease, frailty, neuropsychiatric disorders, secondary malignancies (new cancers caused by treatment), and endocrinopathies.

Suggested MER for Evaluation:

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  Physical examination;

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  Report showing history of HSCT within the past 12 months; and

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  Diagnostic testing and reports related to the underlying condition that was treated with HSCT.

Suggested Listings for Evaluation:

For these listings we generally consider under a disability at least 12 months after the date of transplantation. Any residual impairment(s) that occurs more than 12 months after the date of transplantation should generally be evaluated under the affected body system.

Meets

7.17

13.05 C

13.06 A or B

13.07 B

13.28 A or B

14.07 B

107.17

113.05 C

113.06 A or B

114.07 B

Hematological disorders treated with HSCT meet the requirements of listing 7.17.

Cancers treated with allogeneic HSCT meet the requirements of listing 13.28 A.

Cancers treated with autologous HSCT meet the requirements of 13.28 B.

Hematological disorders treated with HSCT meet the requirements of listing 107.17.

Equals

7.17

107.17

Sickle cell disease (SCD) or beta thalassemia treated with one of three approved cell-based gene therapies (Casgevy, Lyfgenia, or Zynteglo), generally medically equal listing 7.17.

SCD or beta thalassemia treated with one of three approved cell-based gene therapies (Casgevy, Lyfgenia, or Zynteglo), generally medically equal listing 107.17.