Allogeneic Hematopoietic Cell Transplantation for Myelodysplastic Syndromes and Myeloproliferative Neoplasms

Section: Surgery
Effective Date: July 01, 2018
Revised Date: November 13, 2019

Description

Allogeneic Hematopoietic Cell Transplantation

Allogeneic hematopoietic cell transplantation (allo-HCT) involves the intravenous (IV) infusion of allogeneic (donor) stem cells to reestablish hematopoietic function in individuals whose bone marrow or immune system is damaged or defective. They can be harvested from bone marrow, peripheral blood, or umbilical cord blood and placenta shortly after delivery of neonates.

Criteria

Myeloablative allo-HCT may be considered medically necessary as a treatment of:

  • Myelodysplastic syndromes; or
  • Myeloproliferative neoplasms.

Reduced-intensity conditioning allo-HCT may be considered medically necessary in individuals, who for medical reasons would be unable to tolerate a myeloablative conditioning regimen as a treatment of:

  • Myelodysplastic syndromes; or
  • Myeloproliferative neoplasms.

Myeloablative allo-HCT or reduced-intensity conditioning allo-HCT for myelodysplastic syndromes and myeloproliferative neoplasms that do not meet the criteria of this policy is considered experimental/investigational (E/I) and therefore non-covered because the safety and/or effectiveness of this service cannot be established by the available published peer-reviewed literature.

Procedure Codes

38205 38230 38240 S2140 S2142 S2150

Diagnosis Codes

C88.8 C94.40 C94.41 C94.42 C94.6 D46.0 D46.1
D46.20 D46.21 D46.22 D46.4 D46.9 D46.A D46.B
D46.C D46.Z D47.1 D47.Z9

Professional Statements and Societal Positions Guidelines

National Comprehensive Cancer Network (NCCN)-2019

Current National Comprehensive Cancer Networkclinical guidelines for myelodysplastic syndromes (v.1.2020)make the following general recommendation about allo-HCT:

“For individuals who are transplant candidates, an HLA [human leukocyte antigen]-matched sibling, or HLA-matched unrelated donor can be considered. Results with HLA-matched unrelated donors have improved to levels comparable to those obtained with HLA-matched siblings. With the increasing use of cord blood or HLA-haploidentical related donors, HCT has become a viable option for many individuals. High-dose conditioning is typically used for younger individuals, whereas RIC [reduced-intensity conditioning] for HCT is generally the strategy in older individuals.”

American Society for Blood and Marrow Transplantation-2015

The American Society for Blood and Marrow Transplantation (2015) categorized the Indications for HCT into five areas as follows:

  • Standard of care (S), where indication for HCT is well defined and supported by evidence: 
    • This category includes indications that are well defined and are generally supported by evidence in the form of high-quality clinical trials and/or observational studies (e.g., through CIBMTR or EBMT).
  • Standard of care (C), clinical evidence available, where large clinical trials and observational studies are not available but HCT has been shown to be effective therapy: 
    • This category includes indications for which large clinical trials and observational studies are not available. However, HCT has been shown to be an effective therapy with acceptable risk of morbidity and mortality in sufficiently large single- or multi-center cohort studies. HCT can be considered as a treatment option for individual individuals after careful evaluation of risks and benefits. As more evidence becomes available, some indications may be reclassified as “Standard of Care”.
  • Standard of care (R), rare indication, for rare diseases where HCT has demonstrated effectiveness but large clinical trials and observational studies are not feasible: 
    • Indications included in this category are rare diseases for which clinical trials and observational studies with sufficient number of individuals are not currently feasible because of their very low incidence. However, single- or multi-center or registry studies in relatively small cohorts of individuals have shown HCT to be effective treatment with acceptable risks of morbidity and mortality. For individuals with diseases in this category, HCT can be considered as a treatment option for individual individuals after careful evaluation of risks and benefits.
  • Developmental (D), for diseases where pre-clinical and/or early phase clinical studies show HCT to be a promising treatment option: 
    • Developmental indications include diseases where pre-clinical and/or early phase clinical studies show HCT to be a promising treatment option. HCT is best pursued for these indications as part of a clinical trial. As more evidence becomes available, some indications may be reclassified as “Standard of Care, Clinical Evidence Available” or “Standard of Care”.
  • Not generally recommended (N), where available evidence does not support the routine use of HCT: 
    • Transplantation is not currently recommended for these indications where evidence and clinical practice do not support the routine use of HCT. The effectiveness of non-transplant therapies for an earlier phase of a disease does not justify the risks of HCT. Alternatively, a meaningful benefit is not expected from the procedure in individuals with an advanced phase of a disease. However, this recommendation does not preclude investigation of HCT as a potential treatment and transplantation may be pursued for these indications within the context of a clinical trial.

The ASBMT will periodically review these guidelines and will update them as new evidence becomes available.

Links

ND S-209

  1. InterQual® Level of Care Criteria 2019. Acute Care Adult. McKesson Health Solutions, LLC.
  2. Aoki K, Ishikawa T, Ishiyama K, et al. Allogeneic haematopoietic cell transplantation with reduced-intensity conditioning for elderly patients with advanced myelodysplastic syndromes: a nationwide study. Br J Haematol. 2015; 168(3): 463-466.
  3. Basquiera A, Rivas M, Remaggi G, et al. Allogeneic hematopoietic stem cell transplantation in adults with myelodysplastic syndrome: Experience of the Argentinean Group of Bone Marrow Transplantation (GATMO). Hematology. 2016; 21(3): 162-169.
  4. Kroger NM, Deeg JH, Olavarria E, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015;29(11):2126-2133.
  5. National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Myeloproliferative Neoplasms, Version 3.2019.
  6. National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Myelodysplastic Syndromes, Version 1.2020.
  7. Artz AS, Logan B, Zhu X, et al. The prognostic value of serum C-reactive protein, ferritin, and albumin prior to allogeneic transplantation for acute myeloid leukemia and myelodysplastic syndromes. Haematologica. 2016; 101(11):1426-1433.
  8. Yew PY, Alachkar H, Yamaguchi R, et al. Quantitative characterization of T-cell repertoire in allogeneic hematopoietic stem cell transplant recipients. Bone Marrow Transplantation. 2015; 50(9):1227-1234.
  9. Koenecke C, Göhring G, de Wreede LC, et al. Impact of the revised International Prognostic Scoring System, cytogenetics and monosomal karyotype on outcome after allogeneic stem cell transplantation for myelodysplastic syndromes and secondary acute myeloid leukemia evolving from myelodysplastic syndromes: a retrospective multicenter study of the European Society of Blood and Marrow Transplantation. Haematologica. 2015; 100(3):400–408.
  10. Zeng W, Huang L, Meng F, Liu Z, Zhou J, Sun H. Reduced-intensity and myeloablative conditioning allogeneic hematopoietic stem cell transplantation in patients with acute myeloid leukemia and myelodysplastic syndrome: a meta-analysis and systematic review. Int J Clin Exp Med. 2014; 7(11):4357–4368.
  11. Basquiera AL, Pizzi S, Correas AG, et al. Allogeneic hematopoietic stem cell transplantation in pediatric myelodysplastic syndromes: A multicenter experience from Argentina. Pediatr Blood Cancer. 2015; 62(1):153-7.
  12. Symeonidis A, van Biezen A, de WreedeL, et al. Achievement of complete remission predicts outcome of allogeneic haematopoietic stem cell transplantation in patients with chronic myelomonocytic leukaemia. A study of the Chronic Malignancies Working Party of the European Group for Blood and Marrow Transplantation. Br J Haematol. 2015; 171(2); 239-246.
  13. Pohlen M, Groth C, Sauer T, et al. Outcome of allogeneic stem cell transplantation for AML and myelodysplastic syndrome in elderly patients (>60 years). Bone Marrow Transplant. 2016; 51(11); 1441-1448.
  14.  Heidenreich S, Ziagkos D, de Wreede LC, et al. Allogeneic stem cell transplantation for patents age ≥70 years with myelodysplastic syndrome: A retrospective study of the MDS subcommittee of the chronic malignancies working party of the EBMT. Biol Blood Marrow Transplant. 2017; 23(1):44-52.
  15. Damaj G, Mohty M, Robin M, et al. Upfront allogeneic stem cell transplantation after reducedintensity/nonmyeloablative conditioning for patients with myelodysplastic syndrome: a study by the Société Française de Greffe de Moelle et de Thérapie Cellulaire. Biol Blood Marrow Transplant. 2014; 20(9):1349-55.
  16. Gupta V, Malone AK, Hari PN, et al. Reduced-intensity hematopoietic cell transplantation for patients with primary myelofibrosis: A cohort analysis from the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant. 2014; 20(1):89-97.
  17. Kröger N, Giorgino T, Scott BL, et al. Impact of allogeneic stem cell transplantation on survival of patients less than 65 years of age with primary myelofibrosis. Blood. 2015; 125(21):3347–3364.
  18. Majhail NS, Farnia SH, Carpenter PA, et al. Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2015; 21(11):1863–1869.