Disease-Specific Indications and Outcomes

Related Resources

Patient Resources

Other Diseases

Chemotherapy is a common treatment for many patients with the diseases listed below. However, hematopoietic cell transplantation (HCT) may also be indicated.

Plasma Cell Disorders

  • Light Chain Amyloidosis

    Autologous HCT in amyloidosis has shown improved post-transplantation survival, including both early post-transplant mortality and 5-year survival. [1]

  • POEMS syndrome (osteosclerotic myeloma)

    Autologous HCT and high-dose chemotherapy are both treatment options for this rare plasma cell dyscrasia, but longer follow-up is needed to better determine overall survival. [2]

Malignant Diseases

  • Myeloproliferative neoplasms (MPN)

    MPNs are a group of rare disorders, including BCR-ABL-negative myeloproliferative neoplasms, polycythemia vera, and essential thrombocytosis, where diagnosis usually occurs at a young age with a prognosis that depends on disease progression and effectiveness of conservative treatments. MPNs have been treated with conservative strategies over the years, but autologous HCT is an option that can prolong survival. [3, 4, 5]

  • Myelofibrosis (MF)

    Allogeneic HCT may be the only curative treatment option for MF. Risks must be weighed along with the best conditioning regimen for the patient according to disease process and severity. MF is the rare MPN primary myelofibrosis that occurs in older adults with a variable prognosis. [3, 4, 5]

  • Germ cell tumors
    • Ovarian cancer
      Ovarian cancer is a leading cause of death for women in the United States, and research is continuously evolving to show the clear risks and benefits of allogeneic HCT. [6]
    • Testicular cancer
      Male germ cell tumors are on e of the most common curable malignancies among young adult men. Autologous HCT in early relapse and tandem transplantation show outcomes have improved over time. [7]
  • Neuroblastoma
    Autologous HCT has been well established as a treatment option for patients with neuroblastoma. However, allogeneic HCT may provide decreased relapse and prolonged survival, but more research is needed to narrow immunological parameters to reduce disease recurrence in patients with advanced disease. [8, 9]
  • Ewing family of tumors
    This aggressive sarcoma is optimized by chemotherapy or surgical radiation, but several clinical trials have expanded therapy combinations to improve survival. [10]
  • Medulloblastoma
    Medulloblastoma is the most common malignant brain tumor in the pediatric population. Advanced studies of this complex cancer's biology have led to defined treatment regimens and clinical trials involving autologous HCT. [11]

Non-Malignant Diseases

  • Hemophagocytic lymphohistiocytosis (HLH)
    Early donor search is essential for patients newly diagnosed with HLH. Allogeneic HCT is a treatment indicated for recurrent or progressive HLH that may improve outcomes over time, especially with ongoing efforts to optimize pre-transplant treatment. [12]

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.

Transplant Consultation Guidelines: Plasma Cell Disorders

Light Chain Amyloidosis

  •  At diagnosis
  • At progression and/or relapse

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POEMS syndrome (Osteosclerotic Myeloma)

  • At diagnosis

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Transplant Consultation Guidelines: Other Malignant Diseases

Myeloproliferative neoplasms (MPN)

(including BCR-ABL-negative MPNs 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

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Myelofibrosis (MF)

  • DIPSS Intermediate-2 (INT-2) and high-risk disease
  • DIPSS Intermediate-1 (INT-1) with low platelet counts, refractory, red blood cell transfusion dependent, circulating blast cells > 2% complex cytogenetics
  • Metastatic disease at diagnosis
  • Progressive disease while on therapy or relapsed disease

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Germ cell tumors

  • Poor initial response
  • Short initial response

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Neuroblastoma

  • INSS stage 2 or 3 at diagnosis-MYSN 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

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Ewing family of tumors

  • Metastatic disease at diagnosis
  • First relapse or CR2

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Medulloblastoma

  • First relapse or CR2

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Transplant Consultation Guidelines: Other Non-Malignant Diseases

Hemophagocytic lymphohistiocytosis (HLH)

  • At diagnosis

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View complete Referral Timing Guidelines here

References

  1. D’Souza A, Dispenzieri A, Wirk B, et al. Improved Outcomes After Autologous Hematopoietic Cell Transplantation for Light Chain Amyloidosis: A Center for International Blood and Marrow Transplant Research Study. Journal of clinical oncology : official journal of the American Society of Clinical Oncology. 2015;33(32):3741-3749. doi:10.1200/JCO.2015.62.4015. Access
  2. D’Souza A, Lacy M, Gertz M, et al. Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience. Blood. 2012;120(1):56-62. doi:10.1182/blood-2012-04-423178. Access
  3. Tefferi A. How I treat myelofibrosis. Blood. 2011; 117(13): 3494-3504. Access
  4. 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
  5. 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
  6. Bay J-O, Cabrespine-Faugeras A, Tabrizi R, et al. Allogeneic hematopoietic stem cell transplantation in ovarian cancer-the EBMT experience. International journal of cancer. 2010;127(6):1446-1452. doi:10.1002/ijc.25149. https://onlinelibrary.wiley.com/doi/10.1002/ijc.25149  Access
  7. Kilari D, D’Souza A, Fraser R, et al. Autologous Hematopoietic Stem Cell Transplantation for Male Germ Cell Tumors: Improved Outcomes Over 3 Decades. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2019;25(6):1099-1106. doi:10.1016/j.bbmt.2019.02.015. Access
  8. Hale GA, Arora M, Ahn KW. Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience. Bone Marrow Transplant. 2013; 48(8): 1056-1064. Access
  9. 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
  10. 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
  11. Juraschka K, Taylor MD. Medulloblastoma in the age of molecular subgroups: a review. Journal of neurosurgery Pediatrics. 2019;24(4):353-363. doi:10.3171/2019.5.PEDS18381. Access
  12. Seo JJ.  Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: recent advances and controversies.  Blood Res. 2015; 50(3): 131-139.  Access