CurrentBluecross IdahoPolicy 8.01.25

Hematopoietic Cell Transplantation for Autoimmune Diseases

Customize your policy alerts

Sign up for Bluecross Idaho Policy 8.01.25 alerts

Get alerted when Policy 8.01.25 changes without checking for updates manually.

Monitor payer policy activity

Receive email alerts as soon as relevant payer policies are published or updated.

This policy governs coverage of autologous and allogeneic hematopoietic cell transplantation for autoimmune diseases. HCT is investigational for most listed autoimmune diseases, with medically necessary coverage for selected individuals with multiple sclerosis or systemic sclerosis when specified criteria are met.

OpenPayer is powered by Trek Health's payer performance platform. Trek continuously ingests, validates, and normalizes Transparency in Coverage data alongside payer policies and other commercial payer data to create a structured payer intelligence foundation. OpenPayer uses this foundation to deliver personalized search results, dynamically generated policy pages, and tailored policy monitoring based on each user's payers, specialties, billing codes, and areas of interest. The same intelligence powers broader payer performance workflows, including reimbursement benchmarking, contract evaluation, payer negotiations, and financial decision-making.

CurrentBluecross IdahoPolicy 8.01.25

Hematopoietic Cell Transplantation for Autoimmune Diseases

Customize your policy alerts

Sign up for Bluecross Idaho Policy 8.01.25 alerts

Get alerted when Policy 8.01.25 changes without checking for updates manually.

Monitor payer policy activity

Receive email alerts as soon as relevant payer policies are published or updated.

Policy Summary

PayerBluecross Idaho

PolicyHematopoietic Cell Transplantation for Autoimmune Diseases

Policy CodePolicy 8.01.25

Change TypeNo material change

Effective DateSep 1, 2026

Last ReviewMay 28, 2026

Next Review DateNot scheduled

Key ActionDocument multiple sclerosis subtype, relapse and MRI activity, disease-modifying therapy exposure and treatment failure, EDSS score, disease duration, and ambulatory status.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

InvestigationalHCT for most listed autoimmune diseases

18–55Qualifying MS age range

EDSS 2.0–6.5Qualifying MS functional status

<60Qualifying systemic sclerosis age

Coverage Criteria

Investigational autoimmune disease indications

Considered investigational for the following autoimmune diseases:

Listed autoimmune diseases: Autologous or allogeneic HCT is investigational for systemic lupus erythematosus, juvenile idiopathic or rheumatoid arthritis, chronic inflammatory demyelinating polyneuropathy, type 1 diabetes, and other autoimmune diseases not otherwise covered by this policy.

Medically necessary autologous HCT for multiple sclerosis

Autologous HCT for multiple sclerosis is medically necessary when all applicable criteria are met.

Multiple sclerosis coverage

Disease subtype and activity
- Secondary progressive inflammatory activity: For secondary progressive multiple sclerosis, at least one of the following is present: two or more clinical relapses in the previous 12 months; one or more clinical relapses in the previous 24 months plus MRI evidence of disease activity at a different time point; or confirmed EDSS worsening of at least 1 point over the preceding 12 months.
Disease activity despite DMT
- Prior DMT exposure: Failure of at least one disease-modifying therapy at therapeutic doses for an adequate duration; OR documented intolerance to disease-modifying therapy.
- Treatment failure: Continued clinical relapse activity while on DMT; new or enlarging T2-hyperintense or gadolinium-enhancing MRI lesions while on DMT; OR confirmed disability progression with an EDSS increase of at least 1.0 point while on DMT.
High risk for future disability: At least one of the following is present: at least two relapses per year despite treatment; incomplete recovery from relapses with residual deficits; early accumulation of disability with an EDSS increase of at least 1.0 point within 12 months; high T2 lesion burden; gadolinium-enhancing lesions despite DMT; spinal cord involvement on MRI; or poor response to high-efficacy DMT.
Age and disease duration: The individual is 18 to 55 years old and disease duration is no more than 15 years.18–55 years; disease duration ≤15 years
Functional status: EDSS score is 2.0 to 6.5 and the individual is ambulatory, with or without an assistive device.EDSS 2.0–6.5
Medical risk: The individual does not have medical comorbidities that would increase procedural risk.

Medically necessary autologous HCT for systemic sclerosis

Autologous HCT for systemic sclerosis is medically necessary when all criteria are met.

Systemic sclerosis coverage

Core eligibility: The individual is an adult younger than 60 years; disease duration is no more than 5 years; modified Rodnan skin score is greater than 15; internal-organ involvement is present; and cyclophosphamide treatment has been given for less than 6 months.Age <60 years; disease duration ≤5 years; modified Rodnan skin score >15; cyclophosphamide treatment <6 months
Required clinical conditions: There is no active gastric antral vascular ectasia and no policy exclusion criterion is present.
Qualifying internal-organ involvement: Abnormal electrocardiogram; pulmonary involvement shown by DLCo below 80% of predicted, FVC decline greater than 10% in the last 12 months, pulmonary fibrosis, or ground-glass appearance on high-resolution chest CT; OR scleroderma-related renal disease.

Systemic sclerosis evidence sufficient for net health outcome improvement

Evidence assessment for systemic sclerosis:

Supportive evidence population and outcomes: Randomized trials evaluated adults younger than 60 years with disease duration of no more than 5 years, modified Rodnan skin score greater than 15, and internal-organ involvement; patients with severe and irreversible organ involvement were excluded. Compared with cyclophosphamide alone, HCT was associated with higher short-term adverse events and treatment-related mortality but consistent long-term improvements in mortality, skin score, and forced vital capacity. The evidence is sufficient to determine improvement in net health outcome.Age <60 years; disease duration ≤5 years; modified Rodnan skin score >15; internal-organ involvement

This is an evidence description, not an authorization criterion.

Insufficient evidence for several autoimmune diseases

Evidence assessment for diseases other than systemic sclerosis:

Insufficient evidence determinations: The evidence is insufficient to determine improvement in net health outcome for multiple sclerosis, systemic lupus erythematosus, juvenile idiopathic or rheumatoid arthritis, chronic inflammatory demyelinating polyneuropathy, type 1 diabetes, and other autoimmune diseases including Crohn disease, immune cytopenias, and relapsing polychondritis.

Clinical input supports autologous HCT for appropriately selected individuals with relapsing-remitting or active secondary progressive multiple sclerosis, but the evidence review states that evidence remains insufficient to determine improvement in net health outcome.

Clinical input criteria for selected multiple sclerosis patients

Clinical input supported autologous HCT for appropriately selected individuals when all of the following are met.

Selected multiple sclerosis population: The individual has relapsing-remitting multiple sclerosis or secondary progressive multiple sclerosis with ongoing inflammatory disease activity, such as clinical relapses, MRI activity, or confirmed EDSS worsening; disease progression or continued activity despite at least one disease-modifying therapy at therapeutic doses for an adequate duration, or documented intolerance to disease-modifying therapy; high risk for future disability; age 18 to 55 years with disease duration of 15 years or less; and EDSS score of 2.0 to 6.5 with ambulation, with or without an assistive device.Age 18–55 years; disease duration ≤15 years; EDSS 2.0–6.5; ambulatory

These are clinical-input criteria for a selected subgroup; inclusion of clinical input does not imply alignment with the evidence-review conclusion.

AHCT for multiple sclerosis

AHCT is considered medically necessary for multiple sclerosis when all of the following criteria are met.

All medical-necessity requirements

Eligible multiple sclerosis phenotype
- Secondary progressive inflammatory activity: Two or more clinical relapses in the previous 12 months; one or more clinical relapses in the previous 24 months plus MRI evidence of disease activity at a different time point; OR confirmed EDSS worsening over the preceding 12 months.
Disease progression or continued activity despite DMT
- Prior DMT exposure: Failure of at least one DMT at therapeutic doses for an adequate duration; OR documented intolerance to DMT.
- Evidence of treatment failure while on DMT:

Multiple sclerosis aHSCT candidate criteria

The stated criteria describe disease activity, treatment failure, future disability risk, age, disease duration, and functional status.

Core candidate characteristics

Disease progression or continued activity despite DMT: Prior DMT exposure is demonstrated by failure of at least one DMT at therapeutic doses for an adequate duration or documented intolerance, AND treatment failure is demonstrated by continued clinical relapse activity while on DMT, new or enlarging T2-hyperintense or gadolinium-enhancing MRI lesions while on DMT, or confirmed disability progression with an EDSS increase of at least 1.0 point while on DMT.
High risk for future disability: At least one of the following is present: at least two relapses per year despite treatment; incomplete recovery from relapses with residual deficits; early accumulation of disability with an EDSS increase of at least 1.0 point within 12 months; high T2 lesion burden; gadolinium-enhancing lesions despite DMT; spinal cord involvement; or poor response to high-efficacy DMT.
Age, disease duration, and functional status: The individual is between 18 and 55 years old with disease duration of 15 years or less, has an EDSS score between 2.0 and 6.5, and is ambulatory with or without an assistive device.

Autologous HCT for systemic sclerosis should not be considered when cardiac assessment shows LVEF <50%, tricuspid annular plane systolic excursion <1.8 cm, pulmonary artery systolic pressure >40 mm Hg, or mean pulmonary artery pressure >25 mm Hg.

Autologous HCT for systemic sclerosis should not be considered when pulmonary assessment shows DLCo <40% of predicted or FVC <45% of predicted.

Autologous HCT for systemic sclerosis should not be considered when creatinine clearance is <40 mL/minute.

HCT is not recommended for systemic sclerosis when internal-organ involvement is severe and irreversible. The cardiac, pulmonary, and renal measurements identified in the policy are used to determine when organ involvement presents an exclusion to autologous HCT.

Coding and Thresholds

CPT CodesCPT

38204	Management of recipient hematopoietic progenitor cell donor search and cell acquisition.
38205	Blood-derived hematopoietic progenitor cell harvesting for transplantation, per collection, allogeneic
38206	Blood-derived hematopoietic progenitor cell harvesting for transplantation, per collection, autologous.
38207	Transplant preparation of hematopoietic progenitor cells; cryopreservation and storage.
38208	Thawing of previously frozen harvest, per donor.
38209	Thawing of previously frozen harvest with washing, per donor.
38210	Specific cell depletion with harvest, T-cell depletion.
38211	Tumor cell depletion.
38212	Red blood cell removal.
38212 38213	Platelet depletion.

1–10 of 17

1/2

HCPCS CodesHCPCS

Q0083, Q0084, Q0085	Chemotherapy, administer code range.
J9000-J9999	Chemotherapy drug code range.
S2150	Bone marrow or blood-derived peripheral stem-cell harvesting and transplantation, allogeneic or autologous, including pheresis, high-dose chemotherapy, and the number of days of post-transplant care in the global definition.

Investigational for all relevant diagnosesICD-10Experimental

E10.10-E10.9	Type 1 diabetes mellitus code range.
G35	Multiple sclerosis.
M05.10-M06.9	Rheumatoid arthritis code range.
M08.00-M08.99	Juvenile arthritis code range.
M32.0-M32.9	Systemic lupus erythematosus code range.
M34.0-M34.9	Systemic sclerosis [scleroderma] code range.

ICD-10-PCS CodesICD-10

30243G0	Administration, circulatory, transfusion, central vein, percutaneous, autologous, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243X0	Administration, circulatory, transfusion, central vein, percutaneous, autologous, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243Y0	Administration, circulatory, transfusion, central vein, percutaneous, autologous, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243G2	Administration, circulatory, transfusion, central vein, percutaneous, allogeneic related, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243X2	Administration, circulatory, transfusion, central vein, percutaneous, allogeneic related, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243Y2	Administration, circulatory, transfusion, central vein, percutaneous, allogeneic related, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243Y3	Administration, circulatory, transfusion, central vein, percutaneous, allogeneic unrelated, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243G4	Administration, circulatory, transfusion, central vein, percutaneous, allogeneic, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
30243X4	Administration, circulatory, transfusion, central vein, percutaneous, allogeneic, code by substance (bone marrow, cord blood or stem cells, hematopoietic) code list.
07DQ0ZZ	Surgical, lymphatic and hemic systems, extraction, bone marrow, code list.

1–10 of 13

1/2

Multiple sclerosis age threshold

Required age18 to 55 years

Multiple sclerosis disease duration threshold

Maximum disease duration≤15 years

Multiple sclerosis EDSS range

EDSS range2.0 to 6.5

Multiple sclerosis disability progression threshold

Provider Documentation and Action Items

Step Therapy

Prior Disease-Modifying Therapy Required

Before requesting autologous HCT for multiple sclerosis, document prior exposure to disease-modifying therapy, including failure of at least one DMT at therapeutic doses for an adequate duration or documented intolerance.

Include the DMT name, dose and duration, and the clinical reason for discontinuation or determination of treatment failure.

Documentation Required

Document Multiple Sclerosis Clinical Evidence

Document the multiple sclerosis phenotype and evidence supporting medical necessity for autologous HCT. Coverage requires RRMS, or secondary progressive MS with ongoing inflammatory activity, together with progression or continued activity despite DMT.

For secondary progressive MS, document qualifying relapses, MRI activity at the required time points, or confirmed EDSS worsening of at least 1 point over the preceding 12 months.
Document treatment failure while on DMT and high risk for future disability, such as recurrent relapses, residual deficits, disability accumulation, high lesion burden, enhancing lesions, spinal cord involvement, or poor response to high-efficacy DMT.

Background

Hematopoietic cell transplantation uses hematopoietic stem cells to restore bone marrow and immune function after conditioning treatment. In autoimmune disease, the treatment concept is to ablate and reset the immune system to eliminate self-reactive lymphocytes and generate new, self-tolerant lymphocytes, with the goal of inducing sustained remission.

Definitions and Abbreviations

Hematopoietic cell transplantation definition

DefinitionA procedure in which hematopoietic stem cells are intravenously infused to restore bone marrow and immune function after bone-marrow-toxic treatment; cells may come from the recipient or a donor.

Autologous HCT definition

DefinitionHCT using stem cells obtained from the transplant recipient.

Allogeneic HCT definition

DefinitionHCT using stem cells obtained from a donor; donor-recipient HLA compatibility is an important factor in successful transplantation.

Immune-system reset definition

Candidate Selection

Autoimmune disease HCT candidates

HCT candidate criteria are disease-specific.

Covered autoimmune disease candidates: Autologous HCT may be medically necessary for qualifying multiple sclerosis or systemic sclerosis candidates who meet the policy criteria; HCT for other listed autoimmune diseases is investigational.

Selected multiple sclerosis autologous HCT candidate profile

Clinical input identified a subgroup of appropriately selected multiple sclerosis patients for whom autologous HCT was considered clinically meaningful and consistent with generally accepted practice.

Selected multiple sclerosis candidate profile: The individual has relapsing-remitting or secondary progressive multiple sclerosis with ongoing inflammatory disease activity; disease progression or continued activity despite therapeutic DMT or documented intolerance; high risk for future disability; age 18 to 55 years and disease duration of 15 years or less; EDSS score of 2.0 to 6.5; and ambulation, with or without an assistive device.Age 18–55 years; disease duration ≤15 years; EDSS 2.0–6.5; ambulatory

Contraindications and Exclusions

For systemic sclerosis, autologous HCT should be considered only when the disease is rapidly progressing, the prognosis for survival is poor, and internal-organ involvement is not severe or irreversible. Cardiac exclusions include LVEF <50%, tricuspid annular plane systolic excursion <1.8 cm, pulmonary artery systolic pressure >40 mm Hg, or mean pulmonary artery pressure >25 mm Hg. Pulmonary exclusions include DLCo <40% of predicted or FVC <45% of predicted; the renal exclusion is creatinine clearance <40 mL/minute.

For multiple sclerosis, medical comorbidities that would increase procedural risk are an exclusion from medically necessary coverage. The individual must also remain ambulatory, with or without an assistive device, and meet the required functional-status criteria.

Comorbidities that increase the risk of AHCT, or any medical comorbidity that poses excess procedural risk, should be absent. The patients most likely to benefit are described as still ambulatory and without comorbidities that increase transplant risk.

Evaluation Requirements

Documentation Required

Complete systemic sclerosis organ evaluation

Evaluate systemic sclerosis internal-organ involvement before requesting autologous HCT and submit the required cardiac, pulmonary, and renal measurements. The assessment should also identify exclusion thresholds and whether organ involvement is severe or irreversible.

Cardiac: electrocardiogram, LVEF, tricuspid annular plane systolic excursion, pulmonary artery systolic pressure, and mean pulmonary artery pressure
Pulmonary: DLCo, FVC, pulmonary fibrosis, and high-resolution chest CT findings
Renal: scleroderma-related renal disease and creatinine clearance

Documentation Required

Complete multiple sclerosis candidate evaluation

For multiple sclerosis, document the subtype and disease activity, relapse and MRI history, EDSS trajectory and current functional status, DMT exposure and treatment failure, future-disability risk, disease duration, age, and procedural comorbidity risk.

RRMS or secondary progressive MS with qualifying inflammatory activity
Relapses, MRI lesions, or confirmed EDSS worsening

Policy Summary

PayerBluecross Idaho

PolicyHematopoietic Cell Transplantation for Autoimmune Diseases

Policy CodePolicy 8.01.25

Change TypeNo material change

Effective DateSep 1, 2026

Last ReviewMay 28, 2026

Next Review DateNot scheduled

Key ActionDocument multiple sclerosis subtype, relapse and MRI activity, disease-modifying therapy exposure and treatment failure, EDSS score, disease duration, and ambulatory status.

SourceLink

On This Page

DLCo <80% predicted; FVC decline >10% in 12 months

Continued clinical relapse activity; new or enlarging T2-hyperintense or gadolinium-enhancing MRI lesions; OR confirmed disability progression while on DMT.

High risk for future disability: At least one high-risk feature is present: at least two relapses per year despite treatment; incomplete recovery from relapses with residual deficits; early disability accumulation; high T2 lesion burden; gadolinium-enhancing lesions despite DMT; spinal cord involvement; or poor response to high-efficacy DMT.

Age and disease duration: The individual is 18 to 55 years old and disease duration is no more than 15 years.Age 18–55 years; disease duration ≤15 years

Functional status: EDSS score is 2.0 to 6.5 and the individual is ambulatory, with or without an assistive device.EDSS 2.0–6.5; ambulatory

Age 18–55; disease duration ≤15 years; EDSS 2.0–6.5; ambulatory

Overall interpretation of proposed criteria: A patient need not fulfill all proposed criteria; the common theme is uncontrolled disease based on repeated inflammatory events leading to irreversible early damage.

This is explanatory commentary accompanying the proposed criteria.

Disability progressionEDSS increase ≥1.0 point within 12 months

Systemic sclerosis age threshold

Required age<60 years

Systemic sclerosis disease duration threshold

Maximum disease duration≤5 years

Modified Rodnan skin score threshold

Modified Rodnan skin score>15

DLCo exclusion threshold

DLCo exclusion<40% of predicted value

FVC exclusion threshold

FVC exclusion<45% of predicted value

Renal exclusion threshold

Renal exclusionCreatinine clearance <40 mL/minute

ASSIST primary outcome

Primary outcome at 12 monthsDecrease in mRSS of at least 25% for patients with initial mRSS >14, or increase in FVC of more than 10%

ASSIST disease progression

Disease progressionMore than 25% increase in mRSS or decrease of more than 10% in FVC

Clinically significant skin response

Clinically significant skin responseMore than 25% decrease in mRSS

Clinical relapse threshold

High-risk relapse activity≥2 relapses per year despite treatment

Secondary progressive MS relapse activity

Relapse activityTwo or more clinical relapses in the previous 12 months

Secondary progressive MS relapse and MRI activity

Relapse and MRI activityOne or more clinical relapses in the previous 24 months and MRI evidence of disease activity at a different time point

EDSS progression

EDSS progressionConfirmed EDSS worsening or increase of ≥1 point over the preceding 12 months

Candidate age

Candidate age18 to 55 years

Candidate disease duration

Candidate disease duration≤15 years

Candidate functional status

Functional statusEDSS score 2.0 to 6.5 and ambulatory, with or without an assistive device

Documentation Required

Document Systemic Sclerosis Clinical Evidence

For systemic sclerosis, document that all candidate criteria are satisfied before requesting autologous HCT. The disease should be rapidly progressive with poor survival prognosis and meet the policy’s clinical thresholds.

Document adult age under 60 years, disease duration of no more than 5 years, modified Rodnan skin score greater than 15, qualifying internal-organ involvement, fewer than 6 months of cyclophosphamide treatment, no active gastric antral vascular ectasia, and absence of exclusion criteria.
Include objective cardiac, pulmonary, or renal findings supporting the required internal-organ involvement.

Unsupported block type: prior_auth

Denial Risk

Investigational Autoimmune Disease Indications

Do not represent autologous or allogeneic HCT as medically necessary for autoimmune indications listed as investigational under this policy unless a covered indication and all applicable criteria are documented.

Investigational autoimmune disease indications include systemic lupus erythematosus, juvenile idiopathic or rheumatoid arthritis, chronic inflammatory demyelinating polyneuropathy, and type 1 diabetes.
Autologous HCT for systemic sclerosis that does not meet the stated criteria is considered investigational.

Denial Risk

Systemic Sclerosis Criteria Not Met

Confirm that all systemic sclerosis candidate criteria are satisfied before submitting the request. Failure to document any required threshold or exclusion criterion creates a coverage risk.

Required elements include age under 60 years, disease duration of no more than 5 years, modified Rodnan skin score greater than 15, qualifying internal-organ involvement, fewer than 6 months of cyclophosphamide treatment, no active gastric antral vascular ectasia, and no policy exclusion criteria.

Denial Risk

Severe or Irreversible Organ Involvement

Assess and document the severity and reversibility of internal-organ involvement in systemic sclerosis. HCT may be considered for qualifying involvement, but it is not recommended when organ involvement is severe and irreversible.

Potentially qualifying findings include abnormal ECG; DLCo below 80% predicted, FVC decline greater than 10% in 12 months, pulmonary fibrosis, or ground-glass changes on high-resolution CT; or scleroderma-related renal disease.
Do not consider HCT when cardiac thresholds include LVEF below 50%, TAPSE below 1.8 cm, pulmonary artery systolic pressure above 40 mm Hg, or mean pulmonary artery pressure above 25 mm Hg; pulmonary thresholds include DLCo below 40% or FVC below 45%; or renal creatinine clearance is below 40 mL/minute.

Documentation Required

Functional Status or Comorbidity Risk

Document functional status and relevant comorbidity risk when requesting autologous HCT for multiple sclerosis. The individual must meet the required functional thresholds and must not have comorbidities that increase procedural risk.

Document an EDSS score from 2.0 through 6.5 and that the individual is ambulatory, with or without an assistive device.
Identify and address medical comorbidities that could increase the risk of HCT.

Denial Risk

Failure to Meet Candidate Criteria

Requests that do not demonstrate the applicable candidate criteria should not be submitted as medically necessary. Missing evidence of disease activity, treatment failure, functional eligibility, organ-risk assessment, or required systemic sclerosis thresholds may result in noncoverage.

Verify the complete checklist before submission and provide records supporting each criterion.
If the applicable criteria are not met, autologous HCT may be considered investigational rather than medically necessary.

DefinitionThe treatment concept that ablating and resetting the immune system may alter the autoimmune disease process and induce sustained remission.

Autologous HCT mechanism

MechanismIntended to eliminate self-reactive lymphocytes through lymphoablation and generate new, self-tolerant lymphocytes.

Allogeneic HCT mechanism

MechanismMay potentially eliminate genetic susceptibility to an autoimmune disease; evidence for this approach in autoimmune diseases is currently limited.

TRM definition

TRMTreatment-related mortality.

PFS definition

PFSProgression-free survival.

ARR definition

ARRAnnualized relapse rate.

AHSCT definition

AHSCTAutologous hematopoietic stem cell transplantation.

EDSS definition

EDSSExpanded Disability Status Scale.

HCT definition

HCTHematopoietic cell transplantation.

MS definition

MSMultiple sclerosis.

PFS definition

PFSProgression-free survival.

RRMS definition

RRMSRelapsing-remitting multiple sclerosis.

TRM definition

TRMTreatment-related mortality.

OS definition

OSOverall survival.

PFS definition

PFSProgression-free survival.

EFS definition

EFSEvent-free survival.

TRM definition

TRMTreatment-related mortality.

FVC definition

FVCForced vital capacity.

mRSS definition

mRSSModified Rodnan skin score.

HCT definition

HCTHematopoietic cell transplantation.

HCT definition

HCTHematopoietic cell transplantation.

TRM definition

TRMTreatment-related mortality.

HCT abbreviation

AbbreviationHCT means hematopoietic cell transplantation.

OS definition

OSOverall survival.

PFS definition

PFSProgression-free survival.

C-peptide definition

C-peptideC-peptide measures islet cell mass; an increase after HCT indicates preservation of islet cells.

TRM definition

TRMTreatment-related mortality.

HCT definition

HCTHematopoietic cell transplantation.

AHSCT definition

AHSCTAutologous hematopoietic stem cell transplantation.

OS definition

OSOverall survival.

TRM definition

TRMTreatment-related mortality.

PFS definition

PFSProgression-free survival.

QOL definition

QOLQuality of life.

C classification definition

CStandard of care, clinical evidence available: HCT or immune effector cell therapy has demonstrated effectiveness with acceptable morbidity and mortality in sufficiently large single- or multicenter cohort studies; treatment may be considered for individual patients after careful evaluation of risks and benefits.

R classification definition

RStandard of care, rare indication: smaller single-center, multicenter, or registry cohorts have shown effectiveness with acceptable risks; treatment may be considered for individual patients after careful evaluation of risks and benefits.

D classification definition

DDevelopmental: preclinical or early-phase clinical studies suggest HCT or immune effector cell therapy may be promising; treatment is best pursued as part of a clinical trial.

N classification definition

NNot generally recommended: HCT or immune effector cell therapy is not currently recommended because evidence and clinical practice do not support routine use, although investigation may occur in a clinical trial.

HCT definition

HCTHematopoietic cell transplantation.

EDSS definition

EDSSExpanded Disability Status Scale, a measure of neurological disability and functional status.

AHCT definition

AHCTAutologous hematopoietic cell transplantation.

DMT definition

DMTDisease-modifying therapy used to treat multiple sclerosis.

RRMS definition

RRMSRelapsing-remitting multiple sclerosis.

Clinical-input candidate profile; not necessarily the final coverage determination.

Autologous HSCT candidate criteria

AHCT is considered medically necessary for multiple sclerosis when all of the following criteria are met.

AHCT candidate criteria

MS phenotype: Relapsing-remitting multiple sclerosis; OR secondary progressive multiple sclerosis with defined inflammatory activity.
Treatment failure: Prior DMT exposure and evidence of treatment failure while on DMT are both required.
High future-disability risk: At least one specified high-risk feature is present.
Age and disease duration: Age is 18 to 55 years and disease duration is no more than 15 years.18–55 years; disease duration ≤15 years
Functional status: EDSS score is 2.0 to 6.5 and the individual is ambulatory with or without an assistive device.EDSS 2.0–6.5; ambulatory

aHSCT candidate criteria

Candidate selection for aHSCT is based on uncontrolled autoimmune disease, treatment failure, risk of disability, age, disease duration, and functional status.

aHSCT candidate profile: The individual has disease progression or continued activity despite DMT, is at high risk for future disability, is 18 to 55 years old with disease duration of 15 years or less, and has EDSS score of 2.0 to 6.5 while remaining ambulatory with or without an assistive device.Age 18–55; disease duration ≤15 years; EDSS 2.0–6.5; ambulatory

The detailed disease-activity and high-risk alternatives are specified in the coverage criteria. Commentary states that a patient need not fulfill all proposed criteria.

Prior DMT exposure and treatment failure while on DMT

At least one high-risk feature for future disability

Age 18–55 years, disease duration ≤15 years, EDSS 2.0–6.5, and ambulatory status