Disease-Specific Indications and Outcomes

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Other Malignant and Non-Malignant Diseases

Although chemotherapy or other therapies are appropriate for many patients with the diseases listed below, hematopoietic cell transplantation (HCT) may also be indicated in select patient cohorts.

Malignant Diseases

Germ cell tumors

  • Ovarian cancer
  • Testicular cancer

Myeloproliferative neoplasms (MPN) [1,2,3]

(including BCR-ABL-negative myeloproliferative neoplasms, Polycythemia vera, Essential thrombocytosis)

Myelofibrosis (MF)

 

Neuroblastoma [4,5]

Ewing family of tumors [6]

Medulloblastoma

Non-Malignant Diseases

Hemophagocytic lymphohistiocytosis (HLH) [7]

Referral Timing Guidelines

These guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for autologous or allogeneic transplantation. [8]

Transplant Consultation Guidelines: Other Malignant Diseases

Germ cell tumors

  • Poor initial response
  • Short initial remission

Neuroblastoma

  • INSS stage 2 or 3 at diagnosis
    • MYCN amplification (>4x above reference)
  • INSS stage 4 at diagnosis
    • MYCN amplification (>4x above reference)
    • age >18 months at diagnosis
    • age 12-18 months with unfavorable characteristics
  • Metastatic disease at diagnosis
  • Progressive disease while on therapy or relapsed disease

Ewing Family of Tumors

  • Metastatic disease at diagnosis
  • First relapse or CR2

Medulloblastoma

  • First relapse or CR2

Myeloproliferative Neoplasms (MPN)

(including BCR-ABL-negative myeloproliferative neoplasms, myelofibrosis and later stages of polycythemia vera and essential thrombocytosis)

High resolution HLA typing is recommended at diagnosis for all patients

Intermediate- or high-risk disease including:

  • High-risk cytogenetics
  • Poor initial response or at progression

Myelofibrosis (MF)

  • DIPSS Intemediate-2 (INT-2) and high risk disease
  • DIPSS Intermediate-1 (INT-1) with low platelet counts, red blood cell transfusion dependent, complex cytogenetics 
  • High risk driver mutations (ASXL1, EZH2, TET2, IDH1, IDH2, SRSF2, and TP53) or triple negative (lack of a driver mutation such as JAK2, MPL, or CALR) should be considered in decision making

Download as a slide

 

Transplant Consultation Guidelines: Other Non-Malignant Diseases

Hemophagocytic Lymphohistiocytosis (HLH)

  • At diagnosis

Download as a slide
View complete Referral Timing Guidelines

References

  1. Tefferi A. How I treat myelofibrosis. Blood. 2011; 117(13): 3494-3504. Access
  2. Kröger NM, 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. Access
  3. Deeg HJ, et al. Hematopoietic cell transplantation as curative therapy for patients with myelofibrosis: Long-term success in all age groups. Biol Blood Marrow Transplant. 2015; 21(11): 1883-1887. Access
  4. Hale GA, Arora M, Ahn KW. Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience. Bone Marrow Transplant. 2013; 48(8): 1056-1064. Access
  5. Pinto NR, Applebaum MA, Volchenboum SL, et al.  Advances in Risk Classification and Treatment Strategies for Neuroblastoma.  J Clin Oncol. 2015;33:3008-3017.  Access
  6. Gaspar N, Hawkins DS, Dirksen U, et al.  Ewing Sarcoma: Current Management and Future Approaches Through Collaboration.  J Clin Oncol. 2015;33:3036-3046. Access
  7. Seo JJ.  Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: recent advances and controversies.  Blood Res. 2015; 50(3): 131-139.  Access
  8. NMDP/Be The Match and ASBMT Recommended Timing for Transplant Consultation. Download (PDF)